[go: up one dir, main page]

DK3138911T3 - Crispr-baseret genommodifikation og ¿regulering - Google Patents

Crispr-baseret genommodifikation og ¿regulering Download PDF

Info

Publication number
DK3138911T3
DK3138911T3 DK16183724.0T DK16183724T DK3138911T3 DK 3138911 T3 DK3138911 T3 DK 3138911T3 DK 16183724 T DK16183724 T DK 16183724T DK 3138911 T3 DK3138911 T3 DK 3138911T3
Authority
DK
Denmark
Prior art keywords
sequence
rna
cell
lys
leu
Prior art date
Application number
DK16183724.0T
Other languages
English (en)
Inventor
Fuqiang Chen
Gregory D Davis
Original Assignee
Sigma Aldrich Co Llc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=50883989&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=DK3138911(T3) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Sigma Aldrich Co Llc filed Critical Sigma Aldrich Co Llc
Application granted granted Critical
Publication of DK3138911T3 publication Critical patent/DK3138911T3/da

Links

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0048—Eye, e.g. artificial tears
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • A61P27/10—Ophthalmic agents for accommodation disorders, e.g. myopia
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/463—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from amphibians
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K7/00—Peptides having 5 to 20 amino acids in a fully defined sequence; Derivatives thereof
    • C07K7/04—Linear peptides containing only normal peptide links
    • C07K7/06—Linear peptides containing only normal peptide links having 5 to 11 amino acids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/67—General methods for enhancing the expression
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/96—Stabilising an enzyme by forming an adduct or a composition; Forming enzyme conjugates
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y301/00—Hydrolases acting on ester bonds (3.1)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y301/00—Hydrolases acting on ester bonds (3.1)
    • C12Y301/21—Endodeoxyribonucleases producing 5'-phosphomonoesters (3.1.21)
    • C12Y301/21004—Type II site-specific deoxyribonuclease (3.1.21.4)
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/09—Fusion polypeptide containing a localisation/targetting motif containing a nuclear localisation signal
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/10—Fusion polypeptide containing a localisation/targetting motif containing a tag for extracellular membrane crossing, e.g. TAT or VP22
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
    • C07K2319/81—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor containing a Zn-finger domain for DNA binding
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3513—Protein; Peptide
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites
    • Y—GENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y02—TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
    • Y02A—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE
    • Y02A50/00—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE in human health protection, e.g. against extreme weather
    • Y02A50/30—Against vector-borne diseases, e.g. mosquito-borne, fly-borne, tick-borne or waterborne diseases whose impact is exacerbated by climate change

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Medicinal Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Virology (AREA)
  • Immunology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Mycology (AREA)
  • Cell Biology (AREA)
  • Epidemiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Peptides Or Proteins (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)

Claims (17)

1. Fremgangsmåde til modifikation af en kromosomal sekvens i en eukaryotisk celle ved integrering af en donorsekvens, hvilken fremgangsmåde omfatter: a) introduktion i den eukaryotiske celle (i) af mindst én RNA-guidet endonuklease, der omfatter mindst ét nukleært lokaliseringssignal eller én nukleinsyre, der koder for mindst én RNA-guidet endonuklease, der omfatter mindst ét nukleært lokaliseringssignal, hvor den mindst ene RNA-guidede endonuklease er et Clustered Regularly Interspersed Short Palindromic Repeats (CRISPR)/CRISPR-associeret (Cas) (CRISPR/Cas) type II-systemprotein og CRISPR/Cas type II-systemproteinet er et Cas9-protein, (ii) mindst ét guide-RNA eller -DNA, der koder for mindst ét guide-RNA, og (iii) et donorpolynukleotid, der omfatter donorsekvensen, og b) dyrkning af den eukaryotiske celle, således at hvert guide-RNA guider en RNA-guidet endonuklease til et målsted i den kromosomale sekvens, den RNA-guidede endonuklease introducerer et dobbeltstrenget brud ved målstedet, og det dobbeltstrengede brud repareres af en DNA-reparationsproces, således at den kromosomale sekvens modificeres ved indsætning eller substitution af donorsekvensen i den kromosomale sekvens, hvor målstedet i den kromosomale sekvens umiddelbart efterfølges af et tilstødende protospacer-mønster (PAM), fremgangsmåden ikke omfatter en fremgangsmåde til modificering af den genetiske identitet af et menneskes kimlinje, og hvor fremgangsmåden ikke omfatter a fremgangsmåde til behandling af menneske- eller dyrekroppen ved kirurgi eller terapi.
2. Fremgangsmåde ex vivo eller in vitro til modifikation af en kromosomal sekvens i en eukaryotisk celle ved integrering af en donorsekvens, hvilken fremgangsmåde omfatter: a) introduktion i den eukaryotiske celle af (i) mindst én RNA-guidet endonuklease, der omfatter mindst ét nukleært lokaliseringssignal eller én nukleinsyre, der koder for mindst én RNA-guidet endonuklease, der omfatter mindst ét nukleært lokaliseringssignal, hvor den mindst ene RNA-guidede endonuklease er et Clustered Regularly Interspersed Short Palindromic Repeats (CRISPR)/CRISPR-associeret (Cas) (CRISPR/Cas) type II-systemprotein og CRISPR/Cas type II-systemproteinet er et Cas9-protein, (ii) mindst ét guide-RNA eller -DNA, der koder for mindst ét guide-RNA, og (iii) et donorpolynukleotid, der omfatter donorsekvensen, og b) dyrkning af den eukaryotiske celle, således at hvert guide-RNA guider en RNA-guidet endonuklease til et målsted i den kromosomale sekvens, den RNA-guidede endonuklease introducerer et dobbeltstrenget brud ved målstedet, og det dobbeltstrengede brud repareres af en DNA-reparationsproces, således at den kromosomale sekvens modificeres ved indsætning eller substitution af donorsekvensen i den kromosomale sekvens, hvor målstedet i den kromosomale sekvens umiddelbart efterfølges af et tilstødende protospacer-mønster (PAM), og hvor fremgangsmåden ikke omfatter en fremgangsmåde til modificering af den genetiske identitet af et menneskes kimlinje.
3. Fremgangsmåde ifølge et hvilket som helst foregående krav, hvor målstedet er et Rosa26-locus, et HPRT-locus eller et AAVSl-locus.
4. Fremgangsmåde ifølge et hvilket som helst foregående krav, hvor det mindst ene nukleære lokaliseringssignal befinder sig ved endonukleasens C-terminus.
5. Fremgangsmåde ifølge et hvilket som helst foregående krav, hvor hvert guide-RNA omfatter et første område, der er komplementært til målstedet i den kromosomale sekvens.
6. Fremgangsmåde ifølge et hvilket som helst foregående krav, hvor hvert guide-RNA omfatter et andet område, der interagerer med den RNA-guidede endonuklease.
7. Fremgangsmåde ifølge et hvilket som helst foregående krav, hvor donorsekvensen i donorpolynukleotidet har mindst én nukleotidændring i forhold til den kromosomale sekvens nær målstedet i den kromosomale sekvens.
8. Fremgangsmåde ifølge et hvilket som helst foregående krav, hvor donorsekvensen i donorpolynukleotidet er flankeret af sekvenser med i alt væsentligt sekvensidentitet med sekvenser placeret på opstrøms- og nedstrømssiden af målstedet i den kromosomale sekvens.
9. Fremgangsmåde ifølge et hvilket som helst foregående krav, hvor donorpolynukleotidet endvidere omfatter et målspaltningssted, der genkendes af den RNA-guidede endonuklease.
10. Fremgangsmåde ifølge et hvilket som helst foregående krav, hvor nukleinsyren, der koder for den RNA-guidede endonuklease, er mRNA.
11. Fremgangsmåde ifølge et hvilket som helst af kravene 1-9, hvor nukleinsyren, der koder for den RNA-guidede endonuklease, er DNA.
12. Fremgangsmåde ifølge krav 11, hvor DNA’et er en del af en vektor, der endvidere omfatter sekvenskodning af guide-RNA’et.
13. Fremgangsmåde ifølge et hvilket som helst af kravene 1-12, hvor den eukaryotiske celle er en human celle, en ikke-human pattedyrecelle eller et ikke-humant pattedyrsembryo.
14. Fremgangsmåde ifølge et hvilket som helst af kravene 1-12, hvor den eukaryotiske celle er en celle fra et hvirvelløst dyr, en insektcelle, en plantecelle, en gærcelle eller en enkeltcellet eukaryotisk organisme.
15. Fremgangsmåde ifølge krav 14, hvor den eukaryotiske celle er en plantecelle.
16. Fremgangsmåde ifølge et hvilket som helst af kravene 1-15, hvor den eukaryotiske celle er in vitro.
17. Fremgangsmåde ifølge et hvilket som helst foregående krav, hvor det mindst ene guide -RNA er kemisk syntetiseret.
DK16183724.0T 2012-12-06 2013-12-05 Crispr-baseret genommodifikation og ¿regulering DK3138911T3 (da)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201261734256P 2012-12-06 2012-12-06
US201361758624P 2013-01-30 2013-01-30
US201361761046P 2013-02-05 2013-02-05
US201361794422P 2013-03-15 2013-03-15
EP13859964.2A EP2928496B1 (en) 2012-12-06 2013-12-05 Crispr-based genome modification and regulation

Publications (1)

Publication Number Publication Date
DK3138911T3 true DK3138911T3 (da) 2019-01-21

Family

ID=50883989

Family Applications (6)

Application Number Title Priority Date Filing Date
DK18160519.7T DK3363902T3 (da) 2012-12-06 2013-12-05 Crispr-baseret genommodifikation og -regulering
DK16183725.7T DK3138912T3 (da) 2012-12-06 2013-12-05 Crispr-baseret genommodifikation og -regulering
DK16183723.2T DK3138910T3 (da) 2012-12-06 2013-12-05 Crispr-baseret genommodifikation og -regulering
DK13859964T DK2928496T3 (da) 2012-12-06 2013-12-05 Crispr-baseret genommodifikation og -regulering
DK16183724.0T DK3138911T3 (da) 2012-12-06 2013-12-05 Crispr-baseret genommodifikation og ¿regulering
DK18156734T DK3360964T3 (da) 2012-12-06 2013-12-05 Crispr-baseret genommodifikation og -regulering

Family Applications Before (4)

Application Number Title Priority Date Filing Date
DK18160519.7T DK3363902T3 (da) 2012-12-06 2013-12-05 Crispr-baseret genommodifikation og -regulering
DK16183725.7T DK3138912T3 (da) 2012-12-06 2013-12-05 Crispr-baseret genommodifikation og -regulering
DK16183723.2T DK3138910T3 (da) 2012-12-06 2013-12-05 Crispr-baseret genommodifikation og -regulering
DK13859964T DK2928496T3 (da) 2012-12-06 2013-12-05 Crispr-baseret genommodifikation og -regulering

Family Applications After (1)

Application Number Title Priority Date Filing Date
DK18156734T DK3360964T3 (da) 2012-12-06 2013-12-05 Crispr-baseret genommodifikation og -regulering

Country Status (16)

Country Link
US (15) US20160017366A1 (da)
EP (11) EP3138911B1 (da)
JP (6) JP6620018B2 (da)
KR (7) KR102006880B1 (da)
CN (3) CN105142669B (da)
AU (9) AU2013355214B2 (da)
BR (1) BR112015012375A2 (da)
CA (3) CA2977152C (da)
DK (6) DK3363902T3 (da)
ES (6) ES2757808T3 (da)
IL (5) IL300199B2 (da)
LT (4) LT3363902T (da)
PL (6) PL3363902T3 (da)
PT (6) PT3363902T (da)
SG (4) SG10201800585VA (da)
WO (1) WO2014089290A1 (da)

Families Citing this family (441)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2008027558A2 (en) 2006-08-31 2008-03-06 Codon Devices, Inc. Iterative nucleic acid assembly using activation of vector-encoded traits
EP2637780B1 (en) 2010-11-12 2022-02-09 Gen9, Inc. Protein arrays and methods of using and making the same
AU2011338841B2 (en) 2010-11-12 2017-02-16 Gen9, Inc. Methods and devices for nucleic acids synthesis
BR112013024337A2 (pt) 2011-03-23 2017-09-26 Du Pont locus de traço transgênico complexo em uma planta, planta ou semente, método para produzir em uma planta um locus de traço transgênico complexo e construto de expressão
WO2013066438A2 (en) 2011-07-22 2013-05-10 President And Fellows Of Harvard College Evaluation and improvement of nuclease cleavage specificity
AU2012300401B2 (en) 2011-08-26 2018-02-08 Ginkgo Bioworks, Inc. Compositions and methods for high fidelity assembly of nucleic acids
JP2014531908A (ja) 2011-10-14 2014-12-04 プレジデント アンド フェローズ オブ ハーバード カレッジ 構造アッセンブリによる配列決定
ES2991004T3 (es) 2011-12-22 2024-12-02 Harvard College Métodos para la detección de analitos
GB201122458D0 (en) * 2011-12-30 2012-02-08 Univ Wageningen Modified cascade ribonucleoproteins and uses thereof
US9637739B2 (en) 2012-03-20 2017-05-02 Vilnius University RNA-directed DNA cleavage by the Cas9-crRNA complex
US9150853B2 (en) 2012-03-21 2015-10-06 Gen9, Inc. Methods for screening proteins using DNA encoded chemical libraries as templates for enzyme catalysis
EP2841601B1 (en) 2012-04-24 2019-03-06 Gen9, Inc. Methods for sorting nucleic acids and multiplexed preparative in vitro cloning
JP6275120B2 (ja) 2012-04-25 2018-02-07 リジェネロン・ファーマシューティカルズ・インコーポレイテッドＲｅｇｅｎｅｒｏｎ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． 大きい標的化ベクターによるヌクレアーゼ媒介標的化
WO2013163628A2 (en) 2012-04-27 2013-10-31 Duke University Genetic correction of mutated genes
FI3597749T3 (fi) 2012-05-25 2023-10-09 Univ California Menetelmiä ja koostumuksia rna-ohjattua kohde-dna-modifikaatiota varten ja rna-ohjattua transkription modulaatiota varten
US20150225734A1 (en) 2012-06-19 2015-08-13 Regents Of The University Of Minnesota Gene targeting in plants using dna viruses
US20150191719A1 (en) 2012-06-25 2015-07-09 Gen9, Inc. Methods for Nucleic Acid Assembly and High Throughput Sequencing
EP2877213B1 (en) * 2012-07-25 2020-12-02 The Broad Institute, Inc. Inducible dna binding proteins and genome perturbation tools and applications thereof
KR101656236B1 (ko) * 2012-10-23 2016-09-12 주식회사 툴젠 표적 DNA에 특이적인 가이드 RNA 및 Cas 단백질을 암호화하는 핵산 또는 Cas 단백질을 포함하는, 표적 DNA를 절단하기 위한 조성물 및 이의 용도
CN105051204B (zh) 2012-11-16 2023-07-21 波赛伊达治疗学股份有限公司 位点特异性酶和使用方法
PT3363902T (pt) 2012-12-06 2019-12-19 Sigma Aldrich Co Llc Modificação e regulação de genoma baseadas em crispr
EP2931898B1 (en) 2012-12-12 2016-03-09 The Broad Institute, Inc. Engineering and optimization of systems, methods and compositions for sequence manipulation with functional domains
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
EP2931899A1 (en) 2012-12-12 2015-10-21 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions, methods, knock out libraries and applications thereof
ES2576126T3 (es) 2012-12-12 2016-07-05 The Broad Institute, Inc. Modificación por tecnología genética y optimización de sistemas, métodos y composiciones enzimáticas mejorados para la manipulación de secuencias
EP3434776A1 (en) 2012-12-12 2019-01-30 The Broad Institute, Inc. Methods, models, systems, and apparatus for identifying target sequences for cas enzymes or crispr-cas systems for target sequences and conveying results thereof
US20140310830A1 (en) 2012-12-12 2014-10-16 Feng Zhang CRISPR-Cas Nickase Systems, Methods And Compositions For Sequence Manipulation in Eukaryotes
EP4234696A3 (en) 2012-12-12 2023-09-06 The Broad Institute Inc. Crispr-cas component systems, methods and compositions for sequence manipulation
KR20150105956A (ko) 2012-12-12 2015-09-18 더 브로드 인스티튜트, 인코퍼레이티드 서열 조작 및 치료적 적용을 위한 시스템, 방법 및 조성물의 전달, 유전자 조작 및 최적화
EP2932421A1 (en) 2012-12-12 2015-10-21 The Broad Institute, Inc. Methods, systems, and apparatus for identifying target sequences for cas enzymes or crispr-cas systems for target sequences and conveying results thereof
PT2784162E (pt) * 2012-12-12 2015-08-27 Broad Inst Inc Engenharia de sistemas, métodos e composições guia otimizadas para a manipulação de sequências
EP4282970A3 (en) 2012-12-17 2024-01-17 President and Fellows of Harvard College Rna-guided human genome engineering
JP2016507244A (ja) * 2013-02-27 2016-03-10 ヘルムホルツ・ツェントルム・ミュンヒェン・ドイチェス・フォルシュンクスツェントルム・フューア・ゲズントハイト・ウント・ウムベルト（ゲーエムベーハー）Ｈｅｌｍｈｏｌｔｚ Ｚｅｎｔｒｕｍ ＭｕｅｎｃｈｅｎＤｅｕｔｓｃｈｅｓ Ｆｏｒｓｃｈｕｎｇｓｚｅｎｔｒｕｍ ｆｕｅｒ Ｇｅｓｕｎｄｈｅｉｔ ｕｎｄ Ｕｍｗｅｌｔ （ＧｍｂＨ） Ｃａｓ９ヌクレアーゼによる卵母細胞における遺伝子編集
US10138509B2 (en) 2013-03-12 2018-11-27 President And Fellows Of Harvard College Method for generating a three-dimensional nucleic acid containing matrix
AU2014235794A1 (en) 2013-03-14 2015-10-22 Caribou Biosciences, Inc. Compositions and methods of nucleic acid-targeting nucleic acids
US20140273235A1 (en) * 2013-03-15 2014-09-18 Regents Of The University Of Minnesota ENGINEERING PLANT GENOMES USING CRISPR/Cas SYSTEMS
US10760064B2 (en) 2013-03-15 2020-09-01 The General Hospital Corporation RNA-guided targeting of genetic and epigenomic regulatory proteins to specific genomic loci
US20140273230A1 (en) * 2013-03-15 2014-09-18 Sigma-Aldrich Co., Llc Crispr-based genome modification and regulation
KR102874079B1 (ko) * 2013-03-15 2025-10-22 더 제너럴 하스피탈 코포레이션 Rna-안내 게놈 편집을 위해 특이성을 증가시키기 위한 절단된 안내 rna(tru-grnas)의 이용
US9828582B2 (en) 2013-03-19 2017-11-28 Duke University Compositions and methods for the induction and tuning of gene expression
WO2014172470A2 (en) * 2013-04-16 2014-10-23 Whitehead Institute For Biomedical Research Methods of mutating, modifying or modulating nucleic acid in a cell or nonhuman mammal
EP3456831B1 (en) 2013-04-16 2021-07-14 Regeneron Pharmaceuticals, Inc. Targeted modification of rat genome
AU2014265331B2 (en) * 2013-05-15 2019-12-05 Sangamo Therapeutics, Inc. Methods and compositions for treatment of a genetic condition
EP3004349B1 (en) * 2013-05-29 2018-03-28 Cellectis S.A. A method for producing precise dna cleavage using cas9 nickase activity
US20140356956A1 (en) 2013-06-04 2014-12-04 President And Fellows Of Harvard College RNA-Guided Transcriptional Regulation
RU2690935C2 (ru) * 2013-06-04 2019-06-06 Президент Энд Фэллоуз Оф Харвард Коллидж Направляемая рнк регуляция транскрипции
JP7085716B2 (ja) * 2013-06-05 2022-06-17 デューク ユニバーシティ Ｒｎａガイド遺伝子編集及び遺伝子調節
WO2014199358A1 (en) 2013-06-14 2014-12-18 Cellectis Methods for non-transgenic genome editing in plants
EP3725885A1 (en) 2013-06-17 2020-10-21 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions methods, screens and applications thereof
EP3674411A1 (en) 2013-06-17 2020-07-01 The Broad Institute, Inc. Delivery, engineering and optimization of tandem guide systems, methods and compositions for sequence manipulation
JP6738729B2 (ja) * 2013-06-17 2020-08-12 ザ・ブロード・インスティテュート・インコーポレイテッド 分裂終了細胞の疾患および障害をターゲティングおよびモデリングするための系、方法および組成物の送達、エンジニアリングおよび最適化
CA2915795C (en) * 2013-06-17 2021-07-13 The Broad Institute, Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using viral components
CN105492611A (zh) * 2013-06-17 2016-04-13 布罗德研究所有限公司 用于序列操纵的优化的crispr-cas双切口酶系统、方法以及组合物
CN107995927B (zh) 2013-06-17 2021-07-30 布罗德研究所有限公司 用于肝靶向和治疗的crispr-cas系统、载体和组合物的递送与用途
US10011850B2 (en) 2013-06-21 2018-07-03 The General Hospital Corporation Using RNA-guided FokI Nucleases (RFNs) to increase specificity for RNA-Guided Genome Editing
SG10201800213VA (en) * 2013-07-10 2018-02-27 Harvard College Orthogonal cas9 proteins for rna-guided gene regulation and editing
US11306328B2 (en) 2013-07-26 2022-04-19 President And Fellows Of Harvard College Genome engineering
US20150044192A1 (en) 2013-08-09 2015-02-12 President And Fellows Of Harvard College Methods for identifying a target site of a cas9 nuclease
EP3611268A1 (en) 2013-08-22 2020-02-19 E. I. du Pont de Nemours and Company Plant genome modification using guide rna/cas endonuclease systems and methods of use
US9359599B2 (en) 2013-08-22 2016-06-07 President And Fellows Of Harvard College Engineered transcription activator-like effector (TALE) domains and uses thereof
US9388430B2 (en) 2013-09-06 2016-07-12 President And Fellows Of Harvard College Cas9-recombinase fusion proteins and uses thereof
US9737604B2 (en) 2013-09-06 2017-08-22 President And Fellows Of Harvard College Use of cationic lipids to deliver CAS9
US9340799B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College MRNA-sensing switchable gRNAs
EP3988649B1 (en) 2013-09-18 2024-11-27 Kymab Limited Methods, cells and organisms
WO2015065964A1 (en) 2013-10-28 2015-05-07 The Broad Institute Inc. Functional genomics using crispr-cas systems, compositions, methods, screens and applications thereof
WO2015066119A1 (en) 2013-10-30 2015-05-07 North Carolina State University Compositions and methods related to a type-ii crispr-cas system in lactobacillus buchneri
KR20230054509A (ko) 2013-11-07 2023-04-24 에디타스 메디신, 인코포레이티드 지배적인 gRNA를 이용하는 CRISPR-관련 방법 및 조성물
ES2975317T3 (es) 2013-12-11 2024-07-04 Regeneron Pharma Métodos y composiciones para la modificación dirigida de un genoma
MX388127B (es) 2013-12-11 2025-03-19 Regeneron Pharma Metodos y composiciones para la modificacion dirigida de un genoma.
BR112016013201B1 (pt) 2013-12-12 2023-01-31 The Broad Institute, Inc. Uso de uma composição compreendendo um sistema crispr-cas no tratamento de uma doença genética ocular
WO2015089364A1 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Crystal structure of a crispr-cas system, and uses thereof
EP3080271B1 (en) 2013-12-12 2020-02-12 The Broad Institute, Inc. Systems, methods and compositions for sequence manipulation with optimized functional crispr-cas systems
EP3080259B1 (en) 2013-12-12 2023-02-01 The Broad Institute, Inc. Engineering of systems, methods and optimized guide compositions with new architectures for sequence manipulation
JP2017503485A (ja) 2013-12-12 2017-02-02 ザ・ブロード・インスティテュート・インコーポレイテッド 遺伝子産物の発現、構造情報、及び誘導性モジュラーｃａｓ酵素を変更するためのｃｒｉｓｐｒ−ｃａｓ系並びに方法
EP3540051B1 (en) 2013-12-12 2022-08-17 The Broad Institute, Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for hsv and viral diseases and disorders.
SG10201804973TA (en) 2013-12-12 2018-07-30 Broad Inst Inc Compositions and Methods of Use of Crispr-Cas Systems in Nucleotide Repeat Disorders
US11053481B2 (en) 2013-12-12 2021-07-06 President And Fellows Of Harvard College Fusions of Cas9 domains and nucleic acid-editing domains
US10787654B2 (en) * 2014-01-24 2020-09-29 North Carolina State University Methods and compositions for sequence guiding Cas9 targeting
AU2015217208B2 (en) 2014-02-11 2018-08-30 The Regents Of The University Of Colorado, A Body Corporate CRISPR enabled multiplexed genome engineering
KR20160130392A (ko) 2014-02-18 2016-11-11 듀크 유니버시티 바이러스 복제의 불활성화를 위한 조성물 및 그의 제조 및 사용 방법
WO2015134812A1 (en) 2014-03-05 2015-09-11 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating usher syndrome and retinitis pigmentosa
US11141493B2 (en) 2014-03-10 2021-10-12 Editas Medicine, Inc. Compositions and methods for treating CEP290-associated disease
US11339437B2 (en) 2014-03-10 2022-05-24 Editas Medicine, Inc. Compositions and methods for treating CEP290-associated disease
EP3553176A1 (en) 2014-03-10 2019-10-16 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating leber's congenital amaurosis 10 (lca10)
CA2943622A1 (en) * 2014-03-25 2015-10-01 Editas Medicine Inc. Crispr/cas-related methods and compositions for treating hiv infection and aids
EP3122880B1 (en) 2014-03-26 2021-05-05 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating sickle cell disease
EP3540061A1 (en) 2014-04-02 2019-09-18 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating primary open angle glaucoma
CN106460003A (zh) 2014-04-08 2017-02-22 北卡罗来纳州立大学 用于使用crispr相关基因rna引导阻遏转录的方法和组合物
MX2016014066A (es) 2014-04-28 2017-05-03 Recombinetics Inc Edicion de genes multiples en cerdos.
EP3152319A4 (en) * 2014-06-05 2017-12-27 Sangamo BioSciences, Inc. Methods and compositions for nuclease design
BR112016028564A2 (pt) 2014-06-06 2018-01-30 Regeneron Pharma método para modificar um locus-alvo em uma célula.
SG10202002486QA (en) * 2014-06-16 2020-04-29 Univ Johns Hopkins Compositions and methods for the expression of crispr guide rnas using the h1 promoter
EP3157328B1 (en) * 2014-06-17 2021-08-04 Poseida Therapeutics, Inc. A method for directing proteins to specific loci in the genome and uses thereof
MX384887B (es) * 2014-06-23 2025-03-14 Regeneron Pharma Ensamblaje de adn mediado por nucleasa.
SG10201911411YA (en) 2014-06-26 2020-02-27 Regeneron Pharma Methods and compositions for targeted genetic modifications and methods of use
EP3167071B1 (en) * 2014-07-09 2020-10-07 Gen9, Inc. Compositions and methods for site-directed dna nicking and cleaving
WO2016007839A1 (en) 2014-07-11 2016-01-14 President And Fellows Of Harvard College Methods for high-throughput labelling and detection of biological features in situ using microscopy
US11254933B2 (en) * 2014-07-14 2022-02-22 The Regents Of The University Of California CRISPR/Cas transcriptional modulation
AU2015298571B2 (en) 2014-07-30 2020-09-03 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
WO2016021973A1 (ko) * 2014-08-06 2016-02-11 주식회사 툴젠 캄필로박터 제주니 crispr/cas 시스템 유래 rgen을 이용한 유전체 교정
WO2016033298A1 (en) 2014-08-28 2016-03-03 North Carolina State University Novel cas9 proteins and guiding features for dna targeting and genome editing
US10570418B2 (en) 2014-09-02 2020-02-25 The Regents Of The University Of California Methods and compositions for RNA-directed target DNA modification
WO2016040030A1 (en) 2014-09-12 2016-03-17 E. I. Du Pont De Nemours And Company Generation of site-specific-integration sites for complex trait loci in corn and soybean, and methods of use
ES3015369T3 (en) * 2014-10-09 2025-05-05 Seattle Childrens Hospital Dba Seattle Childrens Res Inst Long poly (a) plasmids and methods for introduction of long poly (a) sequences into the plasmid
EP3204513A2 (en) * 2014-10-09 2017-08-16 Life Technologies Corporation Crispr oligonucleotides and gene editing
EP3204496A1 (en) 2014-10-10 2017-08-16 Editas Medicine, Inc. Compositions and methods for promoting homology directed repair
CA2963315A1 (en) 2014-10-15 2016-04-21 Regeneron Pharmaceuticals, Inc. Methods and compositions for generating or maintaining pluripotent cells
US20170306306A1 (en) * 2014-10-24 2017-10-26 Life Technologies Corporation Compositions and Methods for Enhancing Homologous Recombination
GB201418965D0 (da) 2014-10-24 2014-12-10 Ospedale San Raffaele And Fond Telethon
JP6788584B2 (ja) * 2014-10-31 2020-11-25 マサチューセッツ インスティテュート オブ テクノロジー Ｃｒｉｓｐｒについての超並列コンビナトリアル遺伝学
KR102424721B1 (ko) * 2014-11-06 2022-07-25 이 아이 듀폰 디 네모아 앤드 캄파니 Rna-유도 엔도뉴클레아제의 세포 내로의 펩티드 매개성 전달
AU2015342749B2 (en) 2014-11-07 2022-01-27 Editas Medicine, Inc. Methods for improving CRISPR/Cas-mediated genome-editing
US20170369848A1 (en) * 2014-11-11 2017-12-28 Q Therapeutics, Inc. Engineering mesenchymal stem cells using homologous recombination
US10858662B2 (en) 2014-11-19 2020-12-08 Institute For Basic Science Genome editing with split Cas9 expressed from two vectors
CA3176380A1 (en) 2014-11-21 2016-05-26 Regeneron Pharmaceuticals, Inc. Methods and compositions for targeted genetic modification using paired guide rnas
GB201421096D0 (en) 2014-11-27 2015-01-14 Imp Innovations Ltd Genome editing methods
CN107250148B (zh) 2014-12-03 2021-04-16 安捷伦科技有限公司 具有化学修饰的指导rna
AU2015360502A1 (en) 2014-12-10 2017-06-29 Regents Of The University Of Minnesota Genetically modified cells, tissues, and organs for treating disease
EP3889260A1 (en) 2014-12-12 2021-10-06 The Broad Institute, Inc. Protected guide rnas (pgrnas)
EP3234133B1 (en) * 2014-12-18 2020-11-11 Integrated DNA Technologies, Inc. Crispr-based compositions and methods of use
US10196613B2 (en) 2014-12-19 2019-02-05 Regeneron Pharmaceuticals, Inc. Stem cells for modeling type 2 diabetes
BR112017013104A2 (pt) 2014-12-19 2018-05-15 Regeneron Pharma métodos para modificar um locus genômico alvo em uma célula, para intensificar a recombinação homóloga em um locus genômico alvo em uma célula e para produzir uma geração f0 de um animal não humano.
EP3237615B2 (en) 2014-12-24 2023-07-26 The Broad Institute, Inc. Crispr having or associated with destabilization domains
WO2016120480A1 (fr) 2015-01-29 2016-08-04 Meiogenix Procede pour induire des recombinaisons meiotiques ciblees
EP3256487A4 (en) 2015-02-09 2018-07-18 Duke University Compositions and methods for epigenome editing
ES2884838T3 (es) 2015-04-06 2021-12-13 Univ Leland Stanford Junior ARN guía químicamente modificados para la regulación génica mediada por CRISPR/CAS
JP2018522249A (ja) 2015-04-24 2018-08-09 エディタス・メディシン、インコーポレイテッド Ｃａｓ９分子／ガイドｒｎａ分子複合体の評価
EP3289104B1 (en) 2015-04-29 2020-11-04 New York University Method for treating high-grade gliomas
US11845928B2 (en) 2015-05-04 2023-12-19 Tsinghua University Methods and kits for fragmenting DNA
EP3294896A1 (en) 2015-05-11 2018-03-21 Editas Medicine, Inc. Optimized crispr/cas9 systems and methods for gene editing in stem cells
KR20170141217A (ko) * 2015-05-12 2017-12-22 상가모 테라퓨틱스, 인코포레이티드 유전자 발현의 뉴클레아제-매개된 조절
EP3303607A4 (en) 2015-05-29 2018-10-10 North Carolina State University Methods for screening bacteria, archaea, algae, and yeast using crispr nucleic acids
EP3303585A4 (en) 2015-06-03 2018-10-31 Board of Regents of the University of Nebraska Dna editing using single-stranded dna
WO2016201047A1 (en) 2015-06-09 2016-12-15 Editas Medicine, Inc. Crispr/cas-related methods and compositions for improving transplantation
CN107922918B (zh) 2015-06-15 2022-10-21 北卡罗来纳州立大学 用于有效递送核酸和基于rna的抗微生物剂的方法和组合物
DK3310909T3 (da) * 2015-06-17 2021-09-13 Poseida Therapeutics Inc Sammensætninger og fremgangsmåder til at føre proteiner til specifikke loci i genomet
WO2016205759A1 (en) 2015-06-18 2016-12-22 The Broad Institute Inc. Engineering and optimization of systems, methods, enzymes and guide scaffolds of cas9 orthologs and variants for sequence manipulation
CN109536474A (zh) 2015-06-18 2019-03-29 布罗德研究所有限公司 降低脱靶效应的crispr酶突变
US20160376610A1 (en) * 2015-06-24 2016-12-29 Sigma-Aldrich Co. Llc Cell cycle dependent genome regulation and modification
GB2557123B (en) 2015-07-31 2021-11-03 Univ Minnesota Modified cells and methods of therapy
WO2017027910A1 (en) 2015-08-14 2017-02-23 The University Of Sydney Connexin 45 inhibition for therapy
JP6905755B2 (ja) 2015-08-25 2021-07-21 デューク ユニバーシティ Ｒｎａガイド型エンドヌクレアーゼを使用してゲノム工学における特異性を改善する組成物および方法
AU2016316845B2 (en) 2015-08-28 2022-03-10 The General Hospital Corporation Engineered CRISPR-Cas9 nucleases
US9512446B1 (en) 2015-08-28 2016-12-06 The General Hospital Corporation Engineered CRISPR-Cas9 nucleases
US9926546B2 (en) 2015-08-28 2018-03-27 The General Hospital Corporation Engineered CRISPR-Cas9 nucleases
CA2999500A1 (en) 2015-09-24 2017-03-30 Editas Medicine, Inc. Use of exonucleases to improve crispr/cas-mediated genome editing
KR101745863B1 (ko) 2015-09-25 2017-06-12 전남대학교산학협력단 Crispr/cas9 시스템을 이용한 프로히비틴2 유전자 제거용 시발체
KR101795999B1 (ko) 2015-09-25 2017-11-09 전남대학교산학협력단 Crispr/cas9 시스템을 이용한 베타2-마이크로글로불린 유전자 제거용 시발체
WO2017058751A1 (en) 2015-09-28 2017-04-06 North Carolina State University Methods and compositions for sequence specific antimicrobials
EP4089175A1 (en) * 2015-10-13 2022-11-16 Duke University Genome engineering with type i crispr systems in eukaryotic cells
DK3365997T3 (da) 2015-10-22 2021-06-07 Ericsson Telefon Ab L M Fremgangsmåder og indretning i forbindelse med selektiv forstærkning af radiosignaler
US12043852B2 (en) 2015-10-23 2024-07-23 President And Fellows Of Harvard College Evolved Cas9 proteins for gene editing
EP3368054A4 (en) 2015-10-28 2019-07-03 Voyager Therapeutics, Inc. REGULATORY EXPRESSION USING THE ADENO-ASSOCIATED VIRUS (AAV)
JP6882282B2 (ja) 2015-11-03 2021-06-02 プレジデント アンド フェローズ オブ ハーバード カレッジ 三次元核酸含有マトリックスの立体撮像のための方法と装置
US11905521B2 (en) 2015-11-17 2024-02-20 The Chinese University Of Hong Kong Methods and systems for targeted gene manipulation
US10240145B2 (en) * 2015-11-25 2019-03-26 The Board Of Trustees Of The Leland Stanford Junior University CRISPR/Cas-mediated genome editing to treat EGFR-mutant lung cancer
MX2018005377A (es) 2015-11-30 2018-11-09 Univ Duke Dianas terapeuticas para la correccion del gen de la distrofina humana por edicion genica y metodos de uso.
WO2017112620A1 (en) 2015-12-22 2017-06-29 North Carolina State University Methods and compositions for delivery of crispr based antimicrobials
SG10202112024PA (en) 2016-01-11 2021-12-30 Univ Leland Stanford Junior Chimeric proteins and methods of immunotherapy
NZ743983A (en) 2016-01-11 2025-08-29 Univ Leland Stanford Junior Chimeric proteins and methods of regulating gene expression
EP3219799A1 (en) 2016-03-17 2017-09-20 IMBA-Institut für Molekulare Biotechnologie GmbH Conditional crispr sgrna expression
EP3433363A1 (en) 2016-03-25 2019-01-30 Editas Medicine, Inc. Genome editing systems comprising repair-modulating enzyme molecules and methods of their use
EP3433364A1 (en) 2016-03-25 2019-01-30 Editas Medicine, Inc. Systems and methods for treating alpha 1-antitrypsin (a1at) deficiency
EP4047092B1 (en) 2016-04-13 2025-07-30 Editas Medicine, Inc. Cas9 fusion molecules, gene editing systems, and methods of use thereof
WO2017180915A2 (en) 2016-04-13 2017-10-19 Duke University Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use
EP3443085B1 (en) * 2016-04-14 2022-09-14 BOCO Silicon Valley, Inc. Genome editing of human neural stem cells using nucleases
US11499168B2 (en) * 2016-04-25 2022-11-15 Universitat Basel Allele editing and applications thereof
EP4613756A3 (en) 2016-04-25 2025-11-12 President And Fellows Of Harvard College Hybridization chain reaction methods for in situ molecular detection
SG11201809710RA (en) * 2016-05-06 2018-11-29 Juno Therapeutics Inc Genetically engineered cells and methods of making the same
WO2017201476A1 (en) 2016-05-20 2017-11-23 Regeneron Pharmaceuticals, Inc. Methods for breaking immunological tolerance using multiple guide rnas
CA3026321C (en) 2016-06-02 2023-10-03 Sigma-Aldrich Co. Llc Using programmable dna binding proteins to enhance targeted genome modification
JP7436145B2 (ja) * 2016-06-03 2024-02-21 アンスティチュ ナショナル ドゥ ラ サンテ エ ドゥ ラ ルシェルシュ メディカル Ｃａｓ９ヌクレアーゼをコードする核酸の食餌コントロール発現及びその使用
EP3463290A4 (en) * 2016-06-03 2019-10-30 Temple University - Of The Commonwealth System of Higher Education REGULATION OF THE NEGATIVE RECONSTITUTION OF HIV-1 BY GENETIZATION STRATEGY
US10767175B2 (en) 2016-06-08 2020-09-08 Agilent Technologies, Inc. High specificity genome editing using chemically modified guide RNAs
US10337051B2 (en) 2016-06-16 2019-07-02 The Regents Of The University Of California Methods and compositions for detecting a target RNA
US11293021B1 (en) 2016-06-23 2022-04-05 Inscripta, Inc. Automated cell processing methods, modules, instruments, and systems
DK3474669T3 (da) 2016-06-24 2022-06-27 Univ Colorado Regents Fremgangsmåde til generering af stregkodede kombinatoriske biblioteker
US11603533B2 (en) 2016-07-12 2023-03-14 Washington University Incorporation of internal polya-encoded poly-lysine sequence tags and their variations for the tunable control of protein synthesis in bacterial and eukaryotic cells
EP3487523B1 (en) 2016-07-19 2023-09-06 Duke University Therapeutic applications of cpf1-based genome editing
KR20240027874A (ko) 2016-07-28 2024-03-04 리제너론 파마슈티칼스 인코포레이티드 Gpr156 변이체 및 이들의 용도
BR112019001783A2 (pt) 2016-07-29 2019-05-07 Regeneron Pharmaceuticals, Inc. mamífero não humano, e, métodos para produzir o mamífero não humano e de triagem de um composto.
WO2018026976A1 (en) 2016-08-02 2018-02-08 Editas Medicine, Inc. Compositions and methods for treating cep290 associated disease
US11078481B1 (en) 2016-08-03 2021-08-03 KSQ Therapeutics, Inc. Methods for screening for cancer targets
KR20250103795A (ko) 2016-08-03 2025-07-07 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 아데노신 핵염기 편집제 및 그의 용도
CN109804066A (zh) 2016-08-09 2019-05-24 哈佛大学的校长及成员们 可编程cas9-重组酶融合蛋白及其用途
US20190185850A1 (en) * 2016-08-20 2019-06-20 Avellino Lab Usa, Inc. Single guide rna/crispr/cas9 systems, and methods of use thereof
US11542509B2 (en) 2016-08-24 2023-01-03 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
US11078483B1 (en) 2016-09-02 2021-08-03 KSQ Therapeutics, Inc. Methods for measuring and improving CRISPR reagent function
US20180105806A1 (en) * 2016-09-07 2018-04-19 Massachusetts Institute Of Technology Method for rna-guided endonuclease-based dna assembly
CN106636197B (zh) * 2016-09-22 2019-09-03 南京市妇幼保健院 一种定向敲降斑马鱼基因组中多拷贝基因的方法
US20190225974A1 (en) 2016-09-23 2019-07-25 BASF Agricultural Solutions Seed US LLC Targeted genome optimization in plants
CN110023494A (zh) 2016-09-30 2019-07-16 加利福尼亚大学董事会 Rna指导的核酸修饰酶及其使用方法
JP7306696B2 (ja) 2016-09-30 2023-07-11 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア Ｒｎａ誘導型核酸修飾酵素及びその使用方法
WO2018071572A1 (en) * 2016-10-11 2018-04-19 Stemgenics, Inc. Nanoparticles functionalized with gene editing tools and related methods
AU2017342543B2 (en) 2016-10-14 2024-06-27 President And Fellows Of Harvard College AAV delivery of nucleobase editors
KR20240064734A (ko) 2016-10-14 2024-05-13 더 제너럴 하스피탈 코포레이션 후성적으로 조절되는 부위-특이적 뉴클레아제
GB201617559D0 (en) 2016-10-17 2016-11-30 University Court Of The University Of Edinburgh The Swine comprising modified cd163 and associated methods
CN110520530A (zh) 2016-10-18 2019-11-29 明尼苏达大学董事会 肿瘤浸润性淋巴细胞和治疗方法
US20190365806A1 (en) * 2016-11-02 2019-12-05 Universität Basel Immunologically discernible cell surface variants for use in cell therapy
US12286727B2 (en) 2016-12-19 2025-04-29 Editas Medicine, Inc. Assessing nuclease cleavage
KR102551664B1 (ko) * 2016-12-22 2023-07-05 인텔리아 테라퓨틱스, 인크. 알파-1 항트립신 결핍을 치료하기 위한 조성물 및 방법
KR102151065B1 (ko) * 2016-12-23 2020-09-02 기초과학연구원 동물 배아의 염기 교정용 조성물 및 염기 교정 방법
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
US11859219B1 (en) 2016-12-30 2024-01-02 Flagship Pioneering Innovations V, Inc. Methods of altering a target nucleotide sequence with an RNA-guided nuclease and a single guide RNA
EP3565907B1 (en) 2017-01-06 2022-05-04 Editas Medicine, Inc. Methods of assessing nuclease cleavage
KR102712656B1 (ko) 2017-01-23 2024-10-04 리제너론 파마슈티칼스 인코포레이티드 Hsd17b13 변종 및 이것의 용도
WO2018140497A1 (en) 2017-01-25 2018-08-02 Efimov Igor R Apparatus and methods for in vitro preclinical human trials
TW201839136A (zh) 2017-02-06 2018-11-01 瑞士商諾華公司 治療血色素異常症之組合物及方法
CN106978438B (zh) * 2017-02-27 2020-08-28 北京大北农生物技术有限公司 提高同源重组效率的方法
EP3592381A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Cancer vaccine
EP3592853A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Suppression of pain by gene editing
KR20190127797A (ko) 2017-03-10 2019-11-13 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 시토신에서 구아닌으로의 염기 편집제
WO2018170184A1 (en) 2017-03-14 2018-09-20 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
CA3057192A1 (en) 2017-03-23 2018-09-27 President And Fellows Of Harvard College Nucleobase editors comprising nucleic acid programmable dna binding proteins
US11913015B2 (en) 2017-04-17 2024-02-27 University Of Maryland, College Park Embryonic cell cultures and methods of using the same
US11834670B2 (en) 2017-04-19 2023-12-05 Global Life Sciences Solutions Usa Llc Site-specific DNA modification using a donor DNA repair template having tandem repeat sequences
US12058986B2 (en) 2017-04-20 2024-08-13 Egenesis, Inc. Method for generating a genetically modified pig with inactivated porcine endogenous retrovirus (PERV) elements
EP3612551B1 (en) 2017-04-21 2024-09-04 The General Hospital Corporation Variants of cpf1 (cas12a) with altered pam specificity
EP3615672A1 (en) 2017-04-28 2020-03-04 Editas Medicine, Inc. Methods and systems for analyzing guide rna molecules
WO2018209158A2 (en) 2017-05-10 2018-11-15 Editas Medicine, Inc. Crispr/rna-guided nuclease systems and methods
WO2018209320A1 (en) 2017-05-12 2018-11-15 President And Fellows Of Harvard College Aptazyme-embedded guide rnas for use with crispr-cas9 in genome editing and transcriptional activation
WO2018218188A2 (en) 2017-05-25 2018-11-29 The General Hospital Corporation Base editors with improved precision and specificity
CN110997906B (zh) 2017-06-05 2024-05-07 雷杰纳荣制药公司 B4galt1变体及其用途
CN110997908A (zh) 2017-06-09 2020-04-10 爱迪塔斯医药公司 工程化的cas9核酸酶
KR102720855B1 (ko) 2017-06-15 2024-10-25 더 리전트 오브 더 유니버시티 오브 캘리포니아 표적화된 비-바이러스 dna 삽입
US10011849B1 (en) 2017-06-23 2018-07-03 Inscripta, Inc. Nucleic acid-guided nucleases
US9982279B1 (en) 2017-06-23 2018-05-29 Inscripta, Inc. Nucleic acid-guided nucleases
KR20240135035A (ko) 2017-06-27 2024-09-10 리제너론 파마슈티칼스 인코포레이티드 인간화 asgr1 유전자좌를 포함하는 비인간 동물
KR102424850B1 (ko) 2017-06-30 2022-07-22 인스크립타 인코포레이티드 자동 세포 처리 방법, 모듈, 기기 및 시스템
WO2019006418A2 (en) 2017-06-30 2019-01-03 Intima Bioscience, Inc. ADENO-ASSOCIATED VIRAL VECTORS FOR GENE THERAPY
WO2019014230A1 (en) 2017-07-11 2019-01-17 Sigma-Aldrich Co. Llc USE OF DOMAINS OF PROTEINS INTERACTING WITH NUCLEOSOMES TO IMPROVE THE TARGETED MODIFICATION OF THE GENOME
EP3652312A1 (en) 2017-07-14 2020-05-20 Editas Medicine, Inc. Systems and methods for targeted integration and genome editing and detection thereof using integrated priming sites
CN111801345A (zh) 2017-07-28 2020-10-20 哈佛大学的校长及成员们 使用噬菌体辅助连续进化(pace)的进化碱基编辑器的方法和组合物
KR20200033259A (ko) 2017-07-31 2020-03-27 리제너론 파마슈티칼스 인코포레이티드 생체내에서 CRISPR/Cas-매개된 파괴 또는 삭제 및 외인성 도너 핵산과의 CRISPR/Cas-유도된 재조합을 평가하기 위한 방법 및 조성물
KR102780441B1 (ko) 2017-07-31 2025-03-17 리제너론 파마슈티칼스 인코포레이티드 Cas-형질전환 마우스 배아 줄기 세포 및 마우스 및 이것의 용도
US20190032053A1 (en) * 2017-07-31 2019-01-31 Sigma-Aldrich Co. Llc Synthetic guide rna for crispr/cas activator systems
RU2019143568A (ru) 2017-07-31 2021-09-02 Регенерон Фармасьютикалс, Инк. Способы и композиции для оценки crispr/cas-индуцированной рекомбинации с экзогенной донорной нуклеиновой кислотой in vivo
EP3665279B1 (en) * 2017-08-09 2023-07-19 Benson Hill, Inc. Compositions and methods for modifying genomes
US10738327B2 (en) 2017-08-28 2020-08-11 Inscripta, Inc. Electroporation cuvettes for automation
WO2019139645A2 (en) 2017-08-30 2019-07-18 President And Fellows Of Harvard College High efficiency base editors comprising gam
WO2019050948A1 (en) * 2017-09-05 2019-03-14 Regeneron Pharmaceuticals, Inc. ADMINISTRATION OF A GENE EDITION SYSTEM HAVING ONLY ONE RETROVIRAL PARTICLE AND METHODS OF GENERATING AND USING
AU2018328120B2 (en) 2017-09-06 2024-11-14 Regeneron Pharmaceuticals, Inc. Single Immunoglobulin Interleukin-1 Receptor Related (SIGIRR) variants and uses thereof
CA3074682A1 (en) 2017-09-07 2019-03-14 Regeneron Pharmaceuticals, Inc. Solute carrier family 14 member 1 (slc14a1) variants and uses thereof
EP3585162B1 (en) 2017-09-29 2023-08-30 Regeneron Pharmaceuticals, Inc. Rodents comprising a humanized ttr locus and methods of use
AU2018338990B2 (en) 2017-09-30 2022-06-23 Inscripta, Inc. Flow through electroporation instrumentation
CA3082251A1 (en) 2017-10-16 2019-04-25 The Broad Institute, Inc. Uses of adenosine base editors
SG11202002822XA (en) 2017-10-16 2020-04-29 Regeneron Pharma Cornulin (crnn) variants and uses thereof
US11634716B2 (en) 2017-10-16 2023-04-25 University of Pittsburgh—of the Commonwealth System of Higher Education Genetically modified mesenchymal stem cells for use in cardiovascular prosthetics
KR102503130B1 (ko) 2017-10-27 2023-02-24 더 리전트 오브 더 유니버시티 오브 캘리포니아 내인성 t 세포 수용체의 표적화된 대체
WO2019089804A1 (en) 2017-11-01 2019-05-09 The Regents Of The University Of California Casy compositions and methods of use
BR112020008654A2 (pt) 2017-11-01 2020-11-10 The Regents Of The University Of California composições casz e métodos de uso
WO2019089808A1 (en) 2017-11-01 2019-05-09 The Regents Of The University Of California Class 2 crispr/cas compositions and methods of use
WO2019090261A1 (en) 2017-11-03 2019-05-09 University Of Florida Research Foundation, Inc. Methods and compositions for plant pathogen resistance in plants
CN111565566B (zh) 2017-11-10 2022-10-21 瑞泽恩制药公司 包含slc30a8突变的非人动物及使用方法
CN120310773A (zh) * 2017-11-16 2025-07-15 阿斯利康(瑞典)有限公司 用于改善基于Cas9的敲入策略的有效性的组合物和方法
JP7361031B2 (ja) 2017-11-30 2023-10-13 リジェネロン・ファーマシューティカルズ・インコーポレイテッド ヒト化ｔｒｋｂ遺伝子座を含む非ヒト動物
EP3724214A4 (en) 2017-12-15 2021-09-01 The Broad Institute Inc. SYSTEMS AND METHODS FOR PREDICTING REPAIR RESULTS IN GENETIC ENGINEERING
WO2019126578A1 (en) * 2017-12-20 2019-06-27 Poseida Therapeutics, Inc. Compositions and methods for directing proteins to specific loci in the genome
KR102098915B1 (ko) * 2017-12-22 2020-04-09 (주)지플러스 생명과학 키메라 게놈 조작 분자 및 방법
MX2020007466A (es) 2018-01-12 2020-11-12 Basf Se Gen subyacente al qtl de la cantidad de espiguillas por espiga en trigo en el cromosoma 7a.
US12098363B2 (en) * 2018-01-26 2024-09-24 The Children's Medical Center Corporation Targeting BCL11A distal regulatory elements with a CAS9-CAS9 fusion for fetal hemoglobin reinduction
SG11202007382TA (en) * 2018-02-15 2020-08-28 Sigma Aldrich Co Llc Engineered cas9 systems for eukaryotic genome modification
US12084676B2 (en) 2018-02-23 2024-09-10 Pioneer Hi-Bred International, Inc. Cas9 orthologs
SG11202008956XA (en) 2018-03-14 2020-10-29 Editas Medicine Inc Systems and methods for the treatment of hemoglobinopathies
CN111885915B (zh) 2018-03-19 2023-04-28 瑞泽恩制药公司 使用crispr/cas系统对动物进行转录调制
EP3775159A4 (en) 2018-03-29 2022-01-19 Inscripta, Inc. AUTOMATED REGULATION OF CELL GROWTH RATES FOR INDUCTION AND TRANSFORMATION
WO2019200004A1 (en) 2018-04-13 2019-10-17 Inscripta, Inc. Automated cell processing instruments comprising reagent cartridges
CN112334577B (zh) 2018-04-19 2023-10-17 加利福尼亚大学董事会 用于基因编辑的组合物和方法
US10858761B2 (en) 2018-04-24 2020-12-08 Inscripta, Inc. Nucleic acid-guided editing of exogenous polynucleotides in heterologous cells
WO2019209926A1 (en) 2018-04-24 2019-10-31 Inscripta, Inc. Automated instrumentation for production of peptide libraries
US10526598B2 (en) 2018-04-24 2020-01-07 Inscripta, Inc. Methods for identifying T-cell receptor antigens
EP3784292A4 (en) * 2018-04-27 2022-01-19 Seattle Children's Hospital (DBA Seattle Children's Research Institute) THERAPEUTIC GENOME EDITING IN HYPER-IGM SEX-LINKED SYNDROME
WO2019213430A1 (en) * 2018-05-03 2019-11-07 The Board Of Trustees Of The Leland Stanford Junior University Compositions and methods for nicking target dna sequences
CA3098874A1 (en) 2018-05-10 2019-11-14 The Board Of Trustees Of The Leland Stanford Junior University Gene therapy methods and compositions using auxotrophic regulatable cells
WO2019213910A1 (en) * 2018-05-10 2019-11-14 Syngenta Participations Ag Methods and compositions for targeted editing of polynucleotides
US12133884B2 (en) 2018-05-11 2024-11-05 Beam Therapeutics Inc. Methods of substituting pathogenic amino acids using programmable base editor systems
KR20210045360A (ko) 2018-05-16 2021-04-26 신테고 코포레이션 가이드 rna 설계 및 사용을 위한 방법 및 시스템
CN108624622A (zh) * 2018-05-16 2018-10-09 湖南艾佳生物科技股份有限公司 一种基于CRISPR-Cas9系统构建的能分泌小鼠白细胞介素-6的基因工程细胞株
WO2019226953A1 (en) 2018-05-23 2019-11-28 The Broad Institute, Inc. Base editors and uses thereof
EP3575402A1 (en) * 2018-06-01 2019-12-04 Algentech SAS Gene targeting
US11827892B2 (en) 2018-06-07 2023-11-28 The State Of Israel, Ministry Of Agriculture & Rural Development, Agricultural Research Organization (Aro) (Volcani Center) Nucleic acid constructs and methods of using same
US20210337753A1 (en) 2018-06-07 2021-11-04 The State Of Israel, Ministry Of Agriculture & Rural Development, Agricultural Research Organization Methods of regenerating and transforming cannabis
CN112469834A (zh) * 2018-06-25 2021-03-09 生物纳米基因公司 Dna的标记
CA3104856A1 (en) 2018-06-29 2020-01-02 Editas Medicine, Inc. Synthetic guide molecules, compositions and methods relating thereto
AU2019292919A1 (en) 2018-06-30 2021-03-11 Inscripta, Inc. Instruments, modules, and methods for improved detection of edited sequences in live cells
EP3830263A4 (en) * 2018-08-03 2022-05-04 Beam Therapeutics, Inc. Multi-effector nucleobase editors and methods of using same to modify a nucleic acid target sequence
GB201813011D0 (en) 2018-08-10 2018-09-26 Vib Vzw Means and methods for drought tolerance in crops
US10752874B2 (en) 2018-08-14 2020-08-25 Inscripta, Inc. Instruments, modules, and methods for improved detection of edited sequences in live cells
US11142740B2 (en) 2018-08-14 2021-10-12 Inscripta, Inc. Detection of nuclease edited sequences in automated modules and instruments
US10532324B1 (en) 2018-08-14 2020-01-14 Inscripta, Inc. Instruments, modules, and methods for improved detection of edited sequences in live cells
KR102158428B1 (ko) * 2018-08-16 2020-09-22 (주)라트바이오 인위적 뉴클레아제를 생산하는 형질전환 배아 및 형질전환 동물의 제작 방법
US20210246472A1 (en) * 2018-08-21 2021-08-12 Sigma-Aldrich Co. Llc Down-regulation of the cytosolic dna sensor pathway
WO2020081149A2 (en) 2018-08-30 2020-04-23 Inscripta, Inc. Improved detection of nuclease edited sequences in automated modules and instruments
WO2020051562A2 (en) 2018-09-07 2020-03-12 Beam Therapeutics Inc. Compositions and methods for improving base editing
CN109055379B (zh) * 2018-09-10 2022-04-15 石铭 一种转基因鸡输卵管生物反应器的制备方法
KR102121817B1 (ko) * 2018-09-12 2020-06-26 한국화학연구원 Crispr 편집 기술을 이용한 재조합 항원을 발현시키는 벡터 및 이를 동시에 다중 삽입시키는 방법
ES2974200T3 (es) 2018-09-13 2024-06-26 Regeneron Pharma Rata nuligénica para el gen del factor H del complemento como modelo de glomerulopatía C3
US12264330B2 (en) 2018-10-01 2025-04-01 North Carolina State University Recombinant type I CRISPR-Cas system and uses thereof for killing target cells
EP3861120A4 (en) 2018-10-01 2023-08-16 North Carolina State University Recombinant type i crispr-cas system
US12264313B2 (en) 2018-10-01 2025-04-01 North Carolina State University Recombinant type I CRISPR-Cas system and uses thereof for genome modification and alteration of expression
US12203123B2 (en) 2018-10-01 2025-01-21 North Carolina State University Recombinant type I CRISPR-Cas system and uses thereof for screening for variant cells
WO2020076976A1 (en) 2018-10-10 2020-04-16 Readcoor, Inc. Three-dimensional spatial molecular indexing
GB2611929B (en) 2018-10-16 2023-11-22 Blueallele Corp Methods for targeted insertion of DNA in genes
BR112021007301A2 (pt) 2018-10-18 2021-07-27 Intellia Therapeutics, Inc. composições e métodos para expressar fator ix
WO2020086475A1 (en) 2018-10-22 2020-04-30 Inscripta, Inc. Engineered enzymes
US11214781B2 (en) 2018-10-22 2022-01-04 Inscripta, Inc. Engineered enzyme
WO2020086908A1 (en) * 2018-10-24 2020-04-30 The Broad Institute, Inc. Constructs for improved hdr-dependent genomic editing
US12281338B2 (en) 2018-10-29 2025-04-22 The Broad Institute, Inc. Nucleobase editors comprising GeoCas9 and uses thereof
KR20200071198A (ko) 2018-12-10 2020-06-19 네오이뮨텍, 인코퍼레이티드 Nrf2 발현 조절 기반 T 세포 항암면역치료법
AU2019398351A1 (en) 2018-12-14 2021-06-03 Pioneer Hi-Bred International, Inc. Novel CRISPR-Cas systems for genome editing
EP3898947A1 (en) 2018-12-19 2021-10-27 King's College London Immunotherapeutic methods and compositions
KR20210105914A (ko) 2018-12-20 2021-08-27 리제너론 파마슈티칼스 인코포레이티드 뉴클레아제-매개 반복부 팽창
JP2022516919A (ja) * 2019-01-04 2022-03-03 ザ・ユニバーシティ・オブ・シカゴ Ｒｎａをモジュレートするためのシステムおよび方法
US20220136004A1 (en) * 2019-01-11 2022-05-05 Chan Zuckerberg Biohub, Inc. Targeted In Vivo Genome Modification
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
WO2020163396A1 (en) 2019-02-04 2020-08-13 The General Hospital Corporation Adenine dna base editor variants with reduced off-target rna editing
EP3924477A4 (en) * 2019-02-14 2023-03-29 Metagenomi, Inc. ENZYMES WITH RUVC DOMAINS
US10913941B2 (en) 2019-02-14 2021-02-09 Metagenomi Ip Technologies, Llc Enzymes with RuvC domains
BR112021016019A2 (pt) 2019-02-15 2021-10-05 Sigma-Aldrich Co. Llc Sistemas e proteínas de fusão crispr/cas
GB201902277D0 (en) 2019-02-19 2019-04-03 King S College London Therapeutic agents
CN113811607A (zh) 2019-03-07 2021-12-17 加利福尼亚大学董事会 CRISPR-Cas效应子多肽和其使用方法
IL286359B2 (en) 2019-03-18 2024-06-01 Regeneron Pharma Crispr/cas dropout screening platform to reveal genetic vulnerabilities associated with tau aggregation
KR20240126075A (ko) 2019-03-18 2024-08-20 리제너론 파마슈티칼스 인코포레이티드 타우 시딩 또는 응집의 유전적 변형제를 식별하기 위한 CRISPR/Cas 스크리닝 플랫폼
DE112020001306T5 (de) 2019-03-19 2022-01-27 Massachusetts Institute Of Technology Verfahren und zusammensetzungen zur editierung von nukleotidsequenzen
EP3947691A4 (en) 2019-03-25 2022-12-14 Inscripta, Inc. SIMULTANEOUS MULTIPLEX GENOME EDITING IN A YEAST
US11001831B2 (en) 2019-03-25 2021-05-11 Inscripta, Inc. Simultaneous multiplex genome editing in yeast
CN118064502A (zh) 2019-04-03 2024-05-24 瑞泽恩制药公司 用于将抗体编码序列插入到安全港基因座中的方法和组合物
ES2923629T3 (es) 2019-04-04 2022-09-29 Regeneron Pharma Métodos para la introducción sin cicatrices de modificaciones dirigidas en vectores de direccionamiento
AU2020253532B2 (en) 2019-04-04 2024-06-20 Regeneron Pharmaceuticals, Inc. Non-human animals comprising a humanized coagulation factor 12 locus
GB201905360D0 (en) 2019-04-16 2019-05-29 Univ Nottingham Fungal strains, production and uses thereof
US12473543B2 (en) 2019-04-17 2025-11-18 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
CA3138329A1 (en) 2019-05-10 2020-11-19 Basf Se Regulatory nucleic acid molecules for enhancing gene expression in plants
WO2020247452A1 (en) 2019-06-04 2020-12-10 Regeneron Pharmaceuticals, Inc. Non-human animals comprising a humanized ttr locus with a beta-slip mutation and methods of use
CN113939593A (zh) 2019-06-06 2022-01-14 因思科瑞普特公司 用于递归的核酸指导的细胞编辑的处治
SG11202111256XA (en) 2019-06-07 2021-11-29 Regeneron Pharma Non-human animals comprising a humanized albumin locus
CA3137765A1 (en) 2019-06-14 2020-12-17 Regeneron Pharmaceuticals, Inc. Models of tauopathy
US10907125B2 (en) 2019-06-20 2021-02-02 Inscripta, Inc. Flow through electroporation modules and instrumentation
CN114008070A (zh) 2019-06-21 2022-02-01 因思科瑞普特公司 导致大肠杆菌赖氨酸产量增加的全基因组合理设计的突变
US10927385B2 (en) 2019-06-25 2021-02-23 Inscripta, Inc. Increased nucleic-acid guided cell editing in yeast
EP3783104A1 (en) * 2019-08-20 2021-02-24 Kemijski Institut Coiled-coil mediated tethering of crispr-cas and exonucleases for enhanced genome editing
WO2021050398A1 (en) * 2019-09-10 2021-03-18 The Regents Of The University Of California Synthetic lethality screening platform for cells undergoing alt
EP4028530A1 (en) 2019-09-12 2022-07-20 Basf Se Regulatory nucleic acid molecules for enhancing gene expression in plants
WO2021050940A1 (en) 2019-09-13 2021-03-18 Regeneron Pharmaceuticals, Inc. Transcription modulation in animals using crispr/cas systems delivered by lipid nanoparticles
WO2021069387A1 (en) 2019-10-07 2021-04-15 Basf Se Regulatory nucleic acid molecules for enhancing gene expression in plants
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
CN110628825A (zh) * 2019-10-14 2019-12-31 上海捷易生物科技有限公司 一种依赖nhej的报告基因敲入组合物及其使用方法
EP4054651A1 (en) 2019-11-08 2022-09-14 Regeneron Pharmaceuticals, Inc. Crispr and aav strategies for x-linked juvenile retinoschisis therapy
WO2021102059A1 (en) 2019-11-19 2021-05-27 Inscripta, Inc. Methods for increasing observed editing in bacteria
WO2021108363A1 (en) 2019-11-25 2021-06-03 Regeneron Pharmaceuticals, Inc. Crispr/cas-mediated upregulation of humanized ttr allele
US12325861B2 (en) 2019-12-03 2025-06-10 Basf Se Regulatory nucleic acid molecules for enhancing gene expression in plants
US10883095B1 (en) 2019-12-10 2021-01-05 Inscripta, Inc. Mad nucleases
US10704033B1 (en) 2019-12-13 2020-07-07 Inscripta, Inc. Nucleic acid-guided nucleases
KR20220116485A (ko) 2019-12-16 2022-08-23 바스프 아그리컬쳐럴 솔루션즈 시드 유에스 엘엘씨 쌍을 형성한 닉카제를 이용하는 개선된 게놈 편집
CN114829607A (zh) 2019-12-18 2022-07-29 因思科瑞普特公司 用于体内检测核酸引导的核酸酶编辑的细胞的Cascade/dCas3互补测定
US20230059309A1 (en) * 2020-01-09 2023-02-23 Pioneer Hi-Bred International, Inc. Two-step gene swap
US10689669B1 (en) 2020-01-11 2020-06-23 Inscripta, Inc. Automated multi-module cell processing methods, instruments, and systems
WO2021154706A1 (en) 2020-01-27 2021-08-05 Inscripta, Inc. Electroporation modules and instrumentation
JP2023511626A (ja) 2020-01-28 2023-03-20 リジェネロン・ファーマシューティカルズ・インコーポレイテッド ヒト化ｐｎｐｌａ３遺伝子座を含む非ヒト動物および使用方法
WO2021158883A1 (en) 2020-02-07 2021-08-12 Regeneron Pharmaceuticals, Inc. Non-human animals comprising a humanized klkb1 locus and methods of use
WO2021178556A1 (en) 2020-03-04 2021-09-10 Regeneron Pharmaceuticals, Inc. Methods and compositions for sensitization of tumor cells to immune therapy
EP4125348A1 (en) 2020-03-23 2023-02-08 Regeneron Pharmaceuticals, Inc. Non-human animals comprising a humanized ttr locus comprising a v30m mutation and methods of use
KR102794727B1 (ko) 2020-03-31 2025-04-11 메타지노미, 인크. 클래스 ii, 유형 ii crispr 시스템
WO2021202938A1 (en) 2020-04-03 2021-10-07 Creyon Bio, Inc. Oligonucleotide-based machine learning
US20210332388A1 (en) 2020-04-24 2021-10-28 Inscripta, Inc. Compositions, methods, modules and instruments for automated nucleic acid-guided nuclease editing in mammalian cells
JP2023525304A (ja) 2020-05-08 2023-06-15 ザ ブロード インスティテュート，インコーポレーテッド 標的二本鎖ヌクレオチド配列の両鎖同時編集のための方法および組成物
US11787841B2 (en) 2020-05-19 2023-10-17 Inscripta, Inc. Rationally-designed mutations to the thrA gene for enhanced lysine production in E. coli
WO2021263146A2 (en) 2020-06-26 2021-12-30 Regeneron Pharmaceuticals, Inc. Non-human animals comprising a humanized ace2 locus
CN111849986A (zh) * 2020-07-24 2020-10-30 江苏集萃药康生物科技有限公司 一种减少CRISPR-Cas9基因编辑中双链DNA片段串联的方法及其应用
US20220049303A1 (en) 2020-08-17 2022-02-17 Readcoor, Llc Methods and systems for spatial mapping of genetic variants
EP4214314A4 (en) 2020-09-15 2024-10-16 Inscripta, Inc. Crispr editing to embed nucleic acid landing pads into genomes of live cells
US11512297B2 (en) 2020-11-09 2022-11-29 Inscripta, Inc. Affinity tag for recombination protein recruitment
US20240002839A1 (en) 2020-12-02 2024-01-04 Decibel Therapeutics, Inc. Crispr sam biosensor cell lines and methods of use thereof
US20240058390A1 (en) * 2020-12-16 2024-02-22 The Administrators Of The Tulane Educational Fund Wnt+ adipocytes, exosomes from wnt+ adipocytes, and methods of making and using them
US11306298B1 (en) 2021-01-04 2022-04-19 Inscripta, Inc. Mad nucleases
US11332742B1 (en) 2021-01-07 2022-05-17 Inscripta, Inc. Mad nucleases
AU2022210762A1 (en) * 2021-01-22 2023-08-24 Metagenomi, Inc. Novel engineered and chimeric nucleases
WO2022170059A1 (en) 2021-02-05 2022-08-11 Christiana Care Health Services, Inc. Methods of and compositions for reducing gene expression and/or activity
US11884924B2 (en) 2021-02-16 2024-01-30 Inscripta, Inc. Dual strand nucleic acid-guided nickase editing
GB202103131D0 (en) 2021-03-05 2021-04-21 Biosystems Tech Limited Method for preparation of research organisms
CA3218511A1 (en) 2021-05-10 2022-11-17 Sqz Biotechnologies Company Methods for delivering genome editing molecules to the nucleus or cytosol of a cell and uses thereof
WO2022251644A1 (en) 2021-05-28 2022-12-01 Lyell Immunopharma, Inc. Nr4a3-deficient immune cells and uses thereof
JP2024520676A (ja) 2021-06-02 2024-05-24 ライエル・イミュノファーマ・インコーポレイテッド Ｎｒ４ａ３欠損免疫細胞及びその使用
WO2023028348A1 (en) 2021-08-27 2023-03-02 Metagenomi, Inc. Enzymes with ruvc domains
US11884915B2 (en) 2021-09-10 2024-01-30 Agilent Technologies, Inc. Guide RNAs with chemical modification for prime editing
CN118119702A (zh) 2021-10-14 2024-05-31 隆萨销售股份有限公司 用于细胞外囊泡产生的经修饰的生产者细胞
CA3232968A1 (en) 2021-10-14 2023-04-20 Jasper Williams Immune cells having co-expressed shrnas and logic gate systems
CN118251491A (zh) 2021-10-28 2024-06-25 瑞泽恩制药公司 用于敲除C5的CRISPR/Cas相关方法及组合物
EP4426828A1 (en) 2021-11-01 2024-09-11 Tome Biosciences, Inc. Single construct platform for simultaneous delivery of gene editing machinery and nucleic acid cargo
WO2023081847A1 (en) 2021-11-04 2023-05-11 Regeneron Pharmaceuticals, Inc. Non-human animals comprising a modified cacng1 locus
EP4437096A4 (en) 2021-11-24 2025-09-24 Metagenomi Inc ENDONUCLEASE SYSTEMS
CA3238939A1 (en) 2021-12-08 2023-06-15 Gaurang Patel Mutant myocilin disease model and uses thereof
GB202118058D0 (en) 2021-12-14 2022-01-26 Univ Warwick Methods to increase yields in crops
US20230279442A1 (en) 2021-12-15 2023-09-07 Versitech Limited Engineered cas9-nucleases and method of use thereof
US20250049006A1 (en) 2021-12-20 2025-02-13 C/O Regeneron Pharmaceuticals, Inc. Non-human animals comprising humanized ace2 and tmprss loci
CA3244180A1 (en) 2021-12-22 2023-06-29 Tome Biosciences Inc CO-ADMINISTRATION OF A GENE EDITOR CONSTRUCTION AND A PATTERN DONOR
EP4457342A1 (en) 2021-12-29 2024-11-06 Bristol-Myers Squibb Company Generation of landing pad cell lines
US20250230412A1 (en) 2022-02-01 2025-07-17 President And Fellows Of Harvard College Methods and compositions for treating cancer
TW202332767A (zh) 2022-02-02 2023-08-16 美商雷傑納榮製藥公司 用於治療龐貝氏症之抗ＴｆＲ：ＧＡＡ及抗ＣＤ６３：ＧＡＡ插入
EP4475671A1 (en) 2022-02-07 2024-12-18 Regeneron Pharmaceuticals, Inc. Compositions and methods for defining optimal treatment timeframes in lysosomal disease
WO2023205744A1 (en) 2022-04-20 2023-10-26 Tome Biosciences, Inc. Programmable gene insertion compositions
EP4514981A2 (en) 2022-04-29 2025-03-05 Regeneron Pharmaceuticals, Inc. Identification of tissue-specific extragenic safe harbors for gene therapy approaches
WO2023215831A1 (en) 2022-05-04 2023-11-09 Tome Biosciences, Inc. Guide rna compositions for programmable gene insertion
CA3256953A1 (en) 2022-05-09 2023-11-16 Regeneron Pharma VECTORS AND METHODS FOR IN VIVO ANTIBODY PRODUCTION
EP4525892A1 (en) 2022-05-19 2025-03-26 Lyell Immunopharma, Inc. Polynucleotides targeting nr4a3 and uses thereof
WO2023225670A2 (en) 2022-05-20 2023-11-23 Tome Biosciences, Inc. Ex vivo programmable gene insertion
JP2025521154A (ja) 2022-05-31 2025-07-08 リジェネロン・ファーマシューティカルズ・インコーポレイテッド Ｃ９ｏｒｆ７２反復伸長疾患のためのｃｒｉｓｐｒ干渉療法
WO2023235725A2 (en) 2022-05-31 2023-12-07 Regeneron Pharmaceuticals, Inc. Crispr-based therapeutics for c9orf72 repeat expansion disease
WO2023250384A2 (en) * 2022-06-22 2023-12-28 The Regents Of The University Of California Crispr-cas effector polypeptides and methods of use thereof
WO2023250511A2 (en) 2022-06-24 2023-12-28 Tune Therapeutics, Inc. Compositions, systems, and methods for reducing low-density lipoprotein through targeted gene repression
GB2621813A (en) 2022-06-30 2024-02-28 Univ Newcastle Preventing disease recurrence in Mitochondrial replacement therapy
WO2024020587A2 (en) 2022-07-22 2024-01-25 Tome Biosciences, Inc. Pleiopluripotent stem cell programmable gene insertion
KR20250048010A (ko) 2022-07-29 2025-04-07 리제너론 파마슈티칼스 인코포레이티드 변형된 트랜스페린 수용체 좌위를 포함하는 비-인간 동물
JP2025528052A (ja) 2022-07-29 2025-08-26 リジェネロン・ファーマシューティカルズ・インコーポレイテッド 脳及び筋肉へのトランスフェリン受容体（ｔｆｒ）媒介送達のための組成物及び方法
CA3261296A1 (en) 2022-08-05 2024-02-08 Regeneron Pharma TDP-43 VARIANTS RESISTANT TO AGGREGATION
WO2024064958A1 (en) 2022-09-23 2024-03-28 Lyell Immunopharma, Inc. Methods for culturing nr4a-deficient cells
WO2024064952A1 (en) 2022-09-23 2024-03-28 Lyell Immunopharma, Inc. Methods for culturing nr4a-deficient cells overexpressing c-jun
CN120265314A (zh) 2022-09-28 2025-07-04 瑞泽恩制药公司 抗体抗性修饰受体以增强基于细胞的疗法
WO2024077174A1 (en) 2022-10-05 2024-04-11 Lyell Immunopharma, Inc. Methods for culturing nr4a-deficient cells
EP4605524A1 (en) 2022-10-20 2025-08-27 Basf Se Regulatory nucleic acid molecules for enhancing gene expression in plants
CN120693347A (zh) 2022-11-04 2025-09-23 瑞泽恩制药公司 钙电压门控通道辅助亚基γ1(CACNG1)结合蛋白和CACNG1介导的向骨骼肌的递送
EP4619438A2 (en) 2022-11-14 2025-09-24 Regeneron Pharmaceuticals, Inc. Compositions and methods for fibroblast growth factor receptor 3-mediated delivery to astrocytes
WO2024107670A1 (en) 2022-11-16 2024-05-23 Regeneron Pharmaceuticals, Inc. Chimeric proteins comprising membrane bound il-12 with protease cleavable linkers
WO2024137514A1 (en) 2022-12-22 2024-06-27 Synthego Corporation Systems and method for automated oligonucleotide synthesis
WO2024138194A1 (en) 2022-12-22 2024-06-27 Tome Biosciences, Inc. Platforms, compositions, and methods for in vivo programmable gene insertion
US20250339384A1 (en) 2023-01-27 2025-11-06 Regeneron Pharmaceuticals, Inc. Modified rhabdovirus glycoproteins and uses thereof
WO2024234006A1 (en) 2023-05-11 2024-11-14 Tome Biosciences, Inc. Systems, compositions, and methods for targeting liver sinusodial endothelial cells (lsecs)
WO2024259309A1 (en) 2023-06-15 2024-12-19 Regeneron Pharmaceuticals, Inc. Gene therapy for hearing disorders
WO2025006963A1 (en) 2023-06-30 2025-01-02 Regeneron Pharmaceuticals, Inc. Methods and compositions for increasing homology-directed repair
US20250049896A1 (en) 2023-07-28 2025-02-13 Regeneron Pharmaceuticals, Inc. Anti-tfr:acid sphingomyelinase for treatment of acid sphingomyelinase deficiency
WO2025029654A2 (en) 2023-07-28 2025-02-06 Regeneron Pharmaceuticals, Inc. Use of bgh-sv40l tandem polya to enhance transgene expression during unidirectional gene insertion
WO2025029657A2 (en) 2023-07-28 2025-02-06 Regeneron Pharmaceuticals, Inc. Anti-tfr:gaa and anti-cd63:gaa insertion for treatment of pompe disease
WO2025038750A2 (en) 2023-08-14 2025-02-20 President And Fellows Of Harvard College Methods and compositions for treating cancer
WO2025049524A1 (en) 2023-08-28 2025-03-06 Regeneron Pharmaceuticals, Inc. Cxcr4 antibody-resistant modified receptors
WO2025050069A1 (en) 2023-09-01 2025-03-06 Tome Biosciences, Inc. Programmable gene insertion using engineered integration enzymes
GB202314578D0 (en) 2023-09-22 2023-11-08 Univ Manchester Methods of producing homoplasmic modified plants or parts thereof
WO2025122754A1 (en) 2023-12-07 2025-06-12 Regeneron Pharmaceuticals, Inc. Gaa knockout non-human animals
WO2025184567A1 (en) 2024-03-01 2025-09-04 Regeneron Pharmaceuticals, Inc. Methods and compositions for re-dosing aav using anti-cd40 antagonistic antibody to suppress host anti-aav antibody response
WO2025217398A1 (en) 2024-04-10 2025-10-16 Lyell Immunopharma, Inc. Methods for culturing cells with improved culture medium
WO2025224107A1 (en) 2024-04-22 2025-10-30 Basecamp Research Ltd Method and compositions for detecting off-target editing
WO2025224182A2 (en) 2024-04-23 2025-10-30 Basecamp Research Ltd Single construct platform for simultaneous delivery of gene editing machinery and nucleic acid cargo
WO2025235388A1 (en) 2024-05-06 2025-11-13 Regeneron Pharmaceuticals, Inc. Transgene genomic identification by nuclease-mediated long read sequencing

Family Cites Families (168)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4952496A (en) 1984-03-30 1990-08-28 Associated Universities, Inc. Cloning and expression of the gene for bacteriophage T7 RNA polymerase
WO1988008450A1 (en) 1987-05-01 1988-11-03 Birdwell Finlayson Gene therapy for metabolite disorders
US5350689A (en) 1987-05-20 1994-09-27 Ciba-Geigy Corporation Zea mays plants and transgenic Zea mays plants regenerated from protoplasts or protoplast-derived cells
US5767367A (en) 1990-06-23 1998-06-16 Hoechst Aktiengesellschaft Zea mays (L.) with capability of long term, highly efficient plant regeneration including fertile transgenic maize plants having a heterologous gene, and their preparation
US7150982B2 (en) 1991-09-09 2006-12-19 Third Wave Technologies, Inc. RNA detection assays
FR2763797B1 (fr) * 1997-05-30 1999-07-16 Tabacs & Allumettes Ind Cigarette a tres faible taux de goudron presentant un gout de tabac comparable a celui d'une cigarette classique a plus fort taux de goudron
US20040186071A1 (en) 1998-04-13 2004-09-23 Bennett C. Frank Antisense modulation of CD40 expression
US20020182673A1 (en) 1998-05-15 2002-12-05 Genentech, Inc. IL-17 homologous polypedies and therapeutic uses thereof
WO2000046386A2 (en) 1999-02-03 2000-08-10 The Children's Medical Center Corporation Gene repair involving the induction of double-stranded dna cleavage at a chromosomal target site
US8183339B1 (en) * 1999-10-12 2012-05-22 Xigen S.A. Cell-permeable peptide inhibitors of the JNK signal transduction pathway
WO2002026967A2 (en) 2000-09-25 2002-04-04 Thomas Jefferson University Targeted gene correction by single-stranded oligodeoxynucleotides
WO2002034771A2 (en) 2000-10-27 2002-05-02 Chiron Srl Nucleic acids and proteins from streptococcus groups a & b
JP3454818B1 (ja) * 2001-03-16 2003-10-06 直哉 小林 肝臓細胞の増殖方法、該方法により得られる肝臓細胞、およびその用途
ES2566561T3 (es) 2001-07-12 2016-04-13 University Of Massachusetts Producción in vivo de ARN pequeños de interferencia que median el silenciamiento génico
US20060253913A1 (en) 2001-12-21 2006-11-09 Yue-Jin Huang Production of hSA-linked butyrylcholinesterases in transgenic mammals
WO2003087341A2 (en) * 2002-01-23 2003-10-23 The University Of Utah Research Foundation Targeted chromosomal mutagenesis using zinc finger nucleases
US20030232410A1 (en) 2002-03-21 2003-12-18 Monika Liljedahl Methods and compositions for using zinc finger endonucleases to enhance homologous recombination
WO2003087993A2 (en) 2002-04-09 2003-10-23 Beattie Kenneth L Oligonucleotide probes for genosensor chips
WO2003104451A2 (en) * 2002-06-06 2003-12-18 Her Majesty The Queen In Right Of Canada As Represented By The Minister Of Agriculture And Agri-Food Modifying dna recombination and repair
JP2006502748A (ja) 2002-09-05 2006-01-26 カリフォルニア インスティテュート オブ テクノロジー 遺伝子ターゲッティングを誘発するキメラヌクレアーゼの使用方法
DE10260805A1 (de) * 2002-12-23 2004-07-22 Geneart Gmbh Verfahren und Vorrichtung zum Optimieren einer Nucleotidsequenz zur Expression eines Proteins
EP3222715A1 (en) 2003-08-08 2017-09-27 Sangamo BioSciences, Inc. Methods and compositions for targeted cleavage and recombination
WO2005070948A1 (en) 2004-01-23 2005-08-04 Intronn, Inc. Correction of alpha-1-antitrypsin genetic defects using spliceosome mediated rna trans splicing
US7972854B2 (en) 2004-02-05 2011-07-05 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
US20050220796A1 (en) 2004-03-31 2005-10-06 Dynan William S Compositions and methods for modulating DNA repair
US7919277B2 (en) 2004-04-28 2011-04-05 Danisco A/S Detection and typing of bacterial strains
WO2006069064A2 (en) * 2004-12-22 2006-06-29 Nucleonics, Inc. Conserved hbv and hcv sequences useful for gene silencing
US7892224B2 (en) 2005-06-01 2011-02-22 Brainlab Ag Inverse catheter planning
US7534819B2 (en) 2005-06-10 2009-05-19 University Of Washington Compositions and methods for intracellular delivery of biotinylated cargo
US20060282289A1 (en) 2005-06-14 2006-12-14 Healthmatch Solutions, Llc System and method for health care financing
US20100055793A1 (en) * 2005-07-25 2010-03-04 Johns Hopkins University Site-specific modification of the human genome using custom-designed zinc finger nucleases
WO2007014275A2 (en) * 2005-07-26 2007-02-01 Sangamo Biosciences, Inc. Targeted integration and expression of exogenous nucleic acid sequences
US10022457B2 (en) 2005-08-05 2018-07-17 Gholam A. Peyman Methods to regulate polarization and enhance function of cells
EP2325332B1 (en) 2005-08-26 2012-10-31 DuPont Nutrition Biosciences ApS Use of CRISPR associated genes (CAS)
KR100877824B1 (ko) * 2005-11-11 2009-01-12 한국생명공학연구원 Ｅ2ｅｐｆ ｕｃｐ-ｖｈｌ 상호작용 및 그 용도
EP2522747A1 (en) * 2006-03-02 2012-11-14 The Ohio State University MicroRNA expression profile associated with pancreatic cancer
EP1994182B1 (en) 2006-03-15 2019-05-29 Siemens Healthcare Diagnostics Inc. Degenerate nucleobase analogs
CA2647544A1 (en) * 2006-03-28 2007-11-08 Novartis Ag Covalently-linked complexes of hiv tat and env proteins
ES2367661T3 (es) 2006-05-10 2011-11-07 Deinove Proceso de ingeniería cromosómica mediante el uso de un nuevo sistema de reparación del adn.
US9816140B2 (en) 2006-05-19 2017-11-14 Dupont Nutrition Biosciences Aps Tagged microorganisms and methods of tagging
EP2447279B1 (en) 2006-05-25 2014-04-09 Sangamo BioSciences, Inc. Methods and compositions for gene inactivation
AU2007258872A1 (en) 2006-06-16 2007-12-21 Danisco A/S Bacterium
EP2518155B1 (en) * 2006-08-04 2014-07-23 Georgia State University Research Foundation, Inc. Enzyme sensors, methods for preparing and using such sensors, and methods of detecting protease activity
NZ579002A (en) 2007-03-02 2012-03-30 Danisco Cultures with improved phage resistance
GB0806086D0 (en) 2008-04-04 2008-05-14 Ulive Entpr Ltd Dendrimer polymer hybrids
WO2010011961A2 (en) 2008-07-25 2010-01-28 University Of Georgia Research Foundation, Inc. Prokaryotic rnai-like system and methods of use
KR20160015400A (ko) 2008-08-22 2016-02-12 상가모 바이오사이언스 인코포레이티드 표적화된 단일가닥 분할 및 표적화된 통합을 위한 방법 및 조성물
WO2010030963A2 (en) 2008-09-15 2010-03-18 Children's Medical Center Corporation Modulation of bcl11a for treatment of hemoglobinopathies
US20100076057A1 (en) * 2008-09-23 2010-03-25 Northwestern University TARGET DNA INTERFERENCE WITH crRNA
WO2010054108A2 (en) 2008-11-06 2010-05-14 University Of Georgia Research Foundation, Inc. Cas6 polypeptides and methods of use
MX337838B (es) 2008-11-07 2016-03-22 Dupont Nutrition Biosci Aps Secuencias de repetidos palindromicos cortos regularmente intercalados agrupados de bifidobacterias.
WO2010066907A1 (en) 2008-12-12 2010-06-17 Danisco A/S Genetic cluster of strains of streptococcus thermophilus having unique rheological properties for dairy fermentation
WO2010075424A2 (en) 2008-12-22 2010-07-01 The Regents Of University Of California Compositions and methods for downregulating prokaryotic genes
GB0823658D0 (en) 2008-12-30 2009-02-04 Angiomed Ag Stent delivery device
US8392349B2 (en) 2009-02-23 2013-03-05 Shalini Vajjhala Global adaptation atlas and method of creating same
US9834787B2 (en) 2009-04-09 2017-12-05 Sangamo Therapeutics, Inc. Targeted integration into stem cells
AU2010243276B2 (en) 2009-04-30 2016-09-15 Fondazione Telethon Ets Gene vector
JP2013500018A (ja) 2009-07-24 2013-01-07 シグマ−アルドリッチ・カンパニー・リミテッド・ライアビリティ・カンパニー ゲノム編集のための方法
US20120192298A1 (en) * 2009-07-24 2012-07-26 Sigma Aldrich Co. Llc Method for genome editing
CA2769262C (en) 2009-07-28 2019-04-30 Sangamo Biosciences, Inc. Methods and compositions for treating trinucleotide repeat disorders
KR101418355B1 (ko) 2009-10-23 2014-07-11 (주)바이오니아 고밀도 유전자 합성기
DE102009052674B4 (de) 2009-11-12 2012-10-18 Karl Weinhold Verfahren und Vorrichtung zum Verbinden von Doppelmantelrohren
US20110294114A1 (en) 2009-12-04 2011-12-01 Cincinnati Children's Hospital Medical Center Optimization of determinants for successful genetic correction of diseases, mediated by hematopoietic stem cells
CA2783351C (en) 2009-12-10 2021-09-07 Regents Of The University Of Minnesota Tal effector-mediated dna modification
WO2011100058A1 (en) 2010-02-09 2011-08-18 Sangamo Biosciences, Inc. Targeted genomic modification with partially single-stranded donor molecules
US10087431B2 (en) 2010-03-10 2018-10-02 The Regents Of The University Of California Methods of generating nucleic acid fragments
EA024121B9 (ru) 2010-05-10 2017-01-30 Дзе Реджентс Ов Дзе Юниверсити Ов Калифорния Композиции эндорибонуклеаз и способы их использования
CA2798988C (en) 2010-05-17 2020-03-10 Sangamo Biosciences, Inc. Tal-effector (tale) dna-binding polypeptides and uses thereof
EP2392208B1 (en) * 2010-06-07 2016-05-04 Helmholtz Zentrum München Deutsches Forschungszentrum für Gesundheit und Umwelt (GmbH) Fusion proteins comprising a DNA-binding domain of a Tal effector protein and a non-specific cleavage domain of a restriction nuclease and their use
WO2011156430A2 (en) 2010-06-07 2011-12-15 Fred Hutchinson Cancer Research Center Generation and expression of engineered i-onui endonuclease and its homologues and uses thereof
AU2011265733B2 (en) 2010-06-14 2014-04-17 Iowa State University Research Foundation, Inc. Nuclease activity of TAL effector and Foki fusion protein
EP3489359A1 (en) * 2010-07-23 2019-05-29 Sigma Aldrich Co. LLC Genome editing using targeting endonucleases and single-stranded nucleic acids
EP2601611B1 (en) 2010-08-02 2020-12-09 Integrated Dna Technologies, Inc. Methods for predicting stability and melting temperatures of nucleic acid duplexes
EP2630156B1 (en) 2010-10-20 2018-08-22 DuPont Nutrition Biosciences ApS Lactococcus crispr-cas sequences
WO2012069657A1 (en) * 2010-11-26 2012-05-31 Institut Pasteur Identification of a human gyrovirus and applications.
US20120214241A1 (en) 2010-12-22 2012-08-23 Josee Laganiere Zinc finger nuclease modification of leucine rich repeat kinase 2 (lrrk2) mutant fibroblasts and ipscs
KR20120096395A (ko) 2011-02-22 2012-08-30 주식회사 툴젠 뉴클레아제에 의해 유전자 변형된 세포를 농축시키는 방법
WO2012164565A1 (en) 2011-06-01 2012-12-06 Yeda Research And Development Co. Ltd. Compositions and methods for downregulating prokaryotic genes
CA3186126A1 (en) 2011-09-21 2013-03-28 Sangamo Biosciences, Inc. Methods and compositions for regulation of transgene expression
HK1200871A1 (en) * 2011-11-16 2015-08-14 Sangamo Therapeutics, Inc. Modified dna-binding proteins and uses thereof
US8450107B1 (en) 2011-11-30 2013-05-28 The Broad Institute Inc. Nucleotide-specific recognition sequences for designer TAL effectors
GB201122458D0 (en) 2011-12-30 2012-02-08 Univ Wageningen Modified cascade ribonucleoproteins and uses thereof
CN104284669A (zh) 2012-02-24 2015-01-14 弗雷德哈钦森癌症研究中心 治疗血红蛋白病的组合物和方法
BR112014021104B1 (pt) 2012-02-29 2023-03-28 Sangamo Biosciences, Inc Proteína de fusão de ocorrência não natural compreendendo um domínio de ligação de dna de dedo de zinco manipulado que se liga a um gene htt, seu uso, método in vitro de modificação da expressão de um gene htt em uma célula, e método de geração de um sistema modelo para o estudo da doença de huntington
US9637739B2 (en) 2012-03-20 2017-05-02 Vilnius University RNA-directed DNA cleavage by the Cas9-crRNA complex
WO2013141680A1 (en) 2012-03-20 2013-09-26 Vilnius University RNA-DIRECTED DNA CLEAVAGE BY THE Cas9-crRNA COMPLEX
AU2013204327B2 (en) 2012-04-20 2016-09-01 Aviagen Cell transfection method
WO2013160230A1 (en) 2012-04-23 2013-10-31 Bayer Cropscience Nv Targeted genome engineering in plants
JP6275120B2 (ja) 2012-04-25 2018-02-07 リジェネロン・ファーマシューティカルズ・インコーポレイテッドＲｅｇｅｎｅｒｏｎ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． 大きい標的化ベクターによるヌクレアーゼ媒介標的化
UA115875C2 (uk) 2012-05-02 2018-01-10 ДАУ АГРОСАЙЄНСІЗ ЕлЕлСі Рослина помідора зі зменшеною активністю малатдегідрогенази та спосіб її отримання
BR112014027813A2 (pt) 2012-05-07 2017-08-08 Dow Agrosciences Llc métodos e composições para integração de transgenes direcionada mediada por nuclease
US11120889B2 (en) 2012-05-09 2021-09-14 Georgia Tech Research Corporation Method for synthesizing a nuclease with reduced off-site cleavage
FI3597749T3 (fi) * 2012-05-25 2023-10-09 Univ California Menetelmiä ja koostumuksia rna-ohjattua kohde-dna-modifikaatiota varten ja rna-ohjattua transkription modulaatiota varten
KR20150027756A (ko) 2012-05-30 2015-03-12 베일러 칼리지 오브 메디신 Dna 수복, 변경 및 대체를 위한 도구로서의 초나선 미니벡터
WO2013188037A2 (en) 2012-06-11 2013-12-19 Agilent Technologies, Inc Method of adaptor-dimer subtraction using a crispr cas6 protein
CN104540382A (zh) 2012-06-12 2015-04-22 弗·哈夫曼-拉罗切有限公司 用于产生条件性敲除等位基因的方法和组合物
EP2674501A1 (en) 2012-06-14 2013-12-18 Agence nationale de sécurité sanitaire de l'alimentation,de l'environnement et du travail Method for detecting and identifying enterohemorrhagic Escherichia coli
WO2013188638A2 (en) 2012-06-15 2013-12-19 The Regents Of The University Of California Endoribonucleases and methods of use thereof
US20150225734A1 (en) 2012-06-19 2015-08-13 Regents Of The University Of Minnesota Gene targeting in plants using dna viruses
ES2697912T3 (es) 2012-07-11 2019-01-29 Sangamo Therapeutics Inc Métodos y composiciones para el tratamiento de enfermedades monogénicas
WO2014011901A2 (en) 2012-07-11 2014-01-16 Sangamo Biosciences, Inc. Methods and compositions for delivery of biologics
EP2877213B1 (en) 2012-07-25 2020-12-02 The Broad Institute, Inc. Inducible dna binding proteins and genome perturbation tools and applications thereof
EP2880171B1 (en) 2012-08-03 2018-10-03 The Regents of The University of California Methods and compositions for controlling gene expression by rna processing
AU2013308770B2 (en) 2012-08-29 2019-01-17 Sangamo Therapeutics, Inc. Methods and compositions for treatment of a genetic condition
CA2884162C (en) 2012-09-07 2020-12-29 Dow Agrosciences Llc Fad3 performance loci and corresponding target site specific binding proteins capable of inducing targeted breaks
UA119135C2 (uk) 2012-09-07 2019-05-10 ДАУ АГРОСАЙЄНСІЗ ЕлЕлСі Спосіб отримання трансгенної рослини
UA118090C2 (uk) 2012-09-07 2018-11-26 ДАУ АГРОСАЙЄНСІЗ ЕлЕлСі Спосіб інтегрування послідовності нуклеїнової кислоти, що представляє інтерес, у ген fad2 у клітині сої та специфічний для локусу fad2 білок, що зв'язується, здатний індукувати спрямований розрив
EP2906602B1 (en) 2012-10-12 2019-01-16 The General Hospital Corporation Transcription activator-like effector (tale) - lysine-specific demethylase 1 (lsd1) fusion proteins
KR101656236B1 (ko) * 2012-10-23 2016-09-12 주식회사 툴젠 표적 DNA에 특이적인 가이드 RNA 및 Cas 단백질을 암호화하는 핵산 또는 Cas 단백질을 포함하는, 표적 DNA를 절단하기 위한 조성물 및 이의 용도
CA2889502A1 (en) 2012-10-30 2014-05-08 Recombinetics, Inc. Control of sexual maturation in animals
US20150291967A1 (en) 2012-10-31 2015-10-15 Luc Mathis Coupling herbicide resistance with targeted insertion of transgenes in plants
US20140127752A1 (en) 2012-11-07 2014-05-08 Zhaohui Zhou Method, composition, and reagent kit for targeted genomic enrichment
PT3363902T (pt) 2012-12-06 2019-12-19 Sigma Aldrich Co Llc Modificação e regulação de genoma baseadas em crispr
WO2014093479A1 (en) 2012-12-11 2014-06-19 Montana State University Crispr (clustered regularly interspaced short palindromic repeats) rna-guided control of gene regulation
PT2784162E (pt) 2012-12-12 2015-08-27 Broad Inst Inc Engenharia de sistemas, métodos e composições guia otimizadas para a manipulação de sequências
ES2576126T3 (es) 2012-12-12 2016-07-05 The Broad Institute, Inc. Modificación por tecnología genética y optimización de sistemas, métodos y composiciones enzimáticas mejorados para la manipulación de secuencias
EP3434776A1 (en) 2012-12-12 2019-01-30 The Broad Institute, Inc. Methods, models, systems, and apparatus for identifying target sequences for cas enzymes or crispr-cas systems for target sequences and conveying results thereof
US20140310830A1 (en) 2012-12-12 2014-10-16 Feng Zhang CRISPR-Cas Nickase Systems, Methods And Compositions For Sequence Manipulation in Eukaryotes
KR20150105956A (ko) 2012-12-12 2015-09-18 더 브로드 인스티튜트, 인코퍼레이티드 서열 조작 및 치료적 적용을 위한 시스템, 방법 및 조성물의 전달, 유전자 조작 및 최적화
EP4234696A3 (en) 2012-12-12 2023-09-06 The Broad Institute Inc. Crispr-cas component systems, methods and compositions for sequence manipulation
EP2931898B1 (en) 2012-12-12 2016-03-09 The Broad Institute, Inc. Engineering and optimization of systems, methods and compositions for sequence manipulation with functional domains
EP2931899A1 (en) 2012-12-12 2015-10-21 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions, methods, knock out libraries and applications thereof
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
EP2932421A1 (en) 2012-12-12 2015-10-21 The Broad Institute, Inc. Methods, systems, and apparatus for identifying target sequences for cas enzymes or crispr-cas systems for target sequences and conveying results thereof
BR102013032129B1 (pt) 2012-12-13 2022-06-07 Dow Agrosciences Llc Método para identificar a presença de um polinucleotídeo de dna doador exógeno inserido dentro de um único locus genômico eucariótico alvo
EP4282970A3 (en) 2012-12-17 2024-01-17 President and Fellows of Harvard College Rna-guided human genome engineering
JP6583918B2 (ja) 2012-12-27 2019-10-02 キージーン ナムローゼ フェンノートシャップ 植物における遺伝連鎖を解消するための方法
EP3919505B1 (en) 2013-01-16 2023-08-30 Emory University Uses of cas9-nucleic acid complexes
CN103233028B (zh) 2013-01-25 2015-05-13 南京徇齐生物技术有限公司 一种无物种限制无生物安全性问题的真核生物基因打靶方法及螺旋结构dna序列
WO2014127287A1 (en) 2013-02-14 2014-08-21 Massachusetts Institute Of Technology Method for in vivo tergated mutagenesis
JP6475172B2 (ja) 2013-02-20 2019-02-27 リジェネロン・ファーマシューティカルズ・インコーポレイテッドＲｅｇｅｎｅｒｏｎ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． ラットの遺伝子組換え
CA2901676C (en) 2013-02-25 2023-08-22 Sangamo Biosciences, Inc. Methods and compositions for enhancing nuclease-mediated gene disruption
AU2014235794A1 (en) 2013-03-14 2015-10-22 Caribou Biosciences, Inc. Compositions and methods of nucleic acid-targeting nucleic acids
US11332719B2 (en) 2013-03-15 2022-05-17 The Broad Institute, Inc. Recombinant virus and preparations thereof
US9234213B2 (en) 2013-03-15 2016-01-12 System Biosciences, Llc Compositions and methods directed to CRISPR/Cas genomic engineering systems
US20140273230A1 (en) 2013-03-15 2014-09-18 Sigma-Aldrich Co., Llc Crispr-based genome modification and regulation
US20140364333A1 (en) 2013-03-15 2014-12-11 President And Fellows Of Harvard College Methods for Live Imaging of Cells
US10760064B2 (en) 2013-03-15 2020-09-01 The General Hospital Corporation RNA-guided targeting of genetic and epigenomic regulatory proteins to specific genomic loci
KR102874079B1 (ko) 2013-03-15 2025-10-22 더 제너럴 하스피탈 코포레이션 Rna-안내 게놈 편집을 위해 특이성을 증가시키기 위한 절단된 안내 rna(tru-grnas)의 이용
US20140273235A1 (en) 2013-03-15 2014-09-18 Regents Of The University Of Minnesota ENGINEERING PLANT GENOMES USING CRISPR/Cas SYSTEMS
EP2981617B1 (en) 2013-04-04 2023-07-05 President and Fellows of Harvard College Therapeutic uses of genome editing with crispr/cas systems
WO2014165612A2 (en) 2013-04-05 2014-10-09 Dow Agrosciences Llc Methods and compositions for integration of an exogenous sequence within the genome of plants
EP3456831B1 (en) 2013-04-16 2021-07-14 Regeneron Pharmaceuticals, Inc. Targeted modification of rat genome
CN103224947B (zh) 2013-04-28 2015-06-10 陕西师范大学 一种基因打靶系统
EP2994531B1 (en) 2013-05-10 2018-03-28 Sangamo Therapeutics, Inc. Delivery methods and compositions for nuclease-mediated genome engineering
US20140349405A1 (en) 2013-05-22 2014-11-27 Wisconsin Alumni Research Foundation Rna-directed dna cleavage and gene editing by cas9 enzyme from neisseria meningitidis
US9873907B2 (en) 2013-05-29 2018-01-23 Agilent Technologies, Inc. Method for fragmenting genomic DNA using CAS9
US20140356956A1 (en) 2013-06-04 2014-12-04 President And Fellows Of Harvard College RNA-Guided Transcriptional Regulation
CN107995927B (zh) 2013-06-17 2021-07-30 布罗德研究所有限公司 用于肝靶向和治疗的crispr-cas系统、载体和组合物的递送与用途
JP6738729B2 (ja) 2013-06-17 2020-08-12 ザ・ブロード・インスティテュート・インコーポレイテッド 分裂終了細胞の疾患および障害をターゲティングおよびモデリングするための系、方法および組成物の送達、エンジニアリングおよび最適化
EP3725885A1 (en) 2013-06-17 2020-10-21 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions methods, screens and applications thereof
CN105492611A (zh) 2013-06-17 2016-04-13 布罗德研究所有限公司 用于序列操纵的优化的crispr-cas双切口酶系统、方法以及组合物
CA2915795C (en) 2013-06-17 2021-07-13 The Broad Institute, Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using viral components
CN103343120B (zh) 2013-07-04 2015-03-04 中国科学院遗传与发育生物学研究所 一种小麦基因组定点改造方法
CN103382468B (zh) * 2013-07-04 2015-04-29 中国科学院遗传与发育生物学研究所 一种水稻基因组定点改造方法
SG10201800213VA (en) 2013-07-10 2018-02-27 Harvard College Orthogonal cas9 proteins for rna-guided gene regulation and editing
CN103388006B (zh) 2013-07-26 2015-10-28 华东师范大学 一种基因定点突变的构建方法
US10421957B2 (en) 2013-07-29 2019-09-24 Agilent Technologies, Inc. DNA assembly using an RNA-programmable nickase
EP3862434A1 (en) 2013-11-04 2021-08-11 Dow AgroSciences LLC Optimal soybean loci
WO2015066638A2 (en) 2013-11-04 2015-05-07 Dow Agrosciences Llc Optimal maize loci
US10233465B2 (en) 2013-11-04 2019-03-19 Dow Agrosciences Llc Optimal soybean loci
KR20230054509A (ko) 2013-11-07 2023-04-24 에디타스 메디신, 인코포레이티드 지배적인 gRNA를 이용하는 CRISPR-관련 방법 및 조성물
MX388127B (es) 2013-12-11 2025-03-19 Regeneron Pharma Metodos y composiciones para la modificacion dirigida de un genoma.
US9850525B2 (en) 2014-01-29 2017-12-26 Agilent Technologies, Inc. CAS9-based isothermal method of detection of specific DNA sequence
US20150291969A1 (en) 2014-01-30 2015-10-15 Chromatin, Inc. Compositions for reduced lignin content in sorghum and improving cell wall digestibility, and methods of making the same
US20150225801A1 (en) 2014-02-11 2015-08-13 California Institute Of Technology Recording and mapping lineage information and molecular events in individual cells
JP6606088B2 (ja) 2014-02-24 2019-11-13 サンガモ セラピューティクス， インコーポレイテッド ヌクレアーゼ媒介性標的化組み込みのための方法および組成物
AU2015231353B2 (en) 2014-03-18 2020-11-05 Sangamo Therapeutics, Inc. Methods and compositions for regulation of zinc finger protein expression

Also Published As

Publication number Publication date
CA2977152C (en) 2019-04-09
KR101844123B1 (ko) 2018-04-02
CA2977152A1 (en) 2014-06-12
EP3138910A1 (en) 2017-03-08
AU2018229489A1 (en) 2018-10-04
EP3138912A1 (en) 2017-03-08
PL3360964T3 (pl) 2020-03-31
KR20210045515A (ko) 2021-04-26
HK1218389A1 (zh) 2017-02-17
ES2757325T3 (es) 2020-04-28
US20160298125A1 (en) 2016-10-13
ES2653212T3 (es) 2018-02-06
IL291129B1 (en) 2023-03-01
AU2023216829A1 (en) 2023-09-14
SG10201800585VA (en) 2018-02-27
CN108913676B (zh) 2023-11-03
PL3363902T3 (pl) 2020-05-18
KR102145760B1 (ko) 2020-08-19
JP2021101706A (ja) 2021-07-15
CA2891347C (en) 2018-02-27
EP3141604A1 (en) 2017-03-15
US20170191082A1 (en) 2017-07-06
KR102243092B1 (ko) 2021-04-22
US20210079427A1 (en) 2021-03-18
PL3138912T3 (pl) 2019-04-30
KR102006880B1 (ko) 2019-08-02
AU2020273316A1 (en) 2020-12-17
EP3138912B1 (en) 2018-12-05
PT3363902T (pt) 2019-12-19
KR20230070065A (ko) 2023-05-19
AU2013355214B2 (en) 2017-06-15
JP2016502840A (ja) 2016-02-01
AU2018229489B2 (en) 2018-12-06
AU2019201344B2 (en) 2020-09-03
IL257178A (en) 2018-03-29
EP3360964A1 (en) 2018-08-15
US10745716B2 (en) 2020-08-18
US20210207173A1 (en) 2021-07-08
KR20180011351A (ko) 2018-01-31
BR112015012375A2 (pt) 2017-09-26
EP3363902B1 (en) 2019-11-27
US20160298134A1 (en) 2016-10-13
AU2019201344C1 (en) 2020-12-24
AU2020230246A1 (en) 2020-10-01
PT3138911T (pt) 2018-12-28
CA3034794A1 (en) 2014-06-12
KR102676910B1 (ko) 2024-06-19
AU2020230243B2 (en) 2021-10-21
ES2714154T3 (es) 2019-05-27
US20170073705A1 (en) 2017-03-16
LT3138910T (lt) 2017-11-10
AU2020230243A1 (en) 2020-10-01
PT2928496T (pt) 2019-11-11
CN108715602A (zh) 2018-10-30
EP3617309A2 (en) 2020-03-04
CN105142669B (zh) 2018-07-03
IL300199B1 (en) 2025-03-01
KR20150091052A (ko) 2015-08-07
US20160298137A1 (en) 2016-10-13
DK3138910T3 (da) 2017-10-16
DK3363902T3 (da) 2020-01-02
AU2022200330B2 (en) 2023-11-09
KR102479178B1 (ko) 2022-12-19
AU2020273316B2 (en) 2023-05-18
EP3135765A1 (en) 2017-03-01
EP3617309A3 (en) 2020-05-06
IL238856A0 (en) 2015-06-30
EP3138911A1 (en) 2017-03-08
IL257178B (en) 2019-07-31
EP3138911B1 (en) 2018-12-05
DK3138912T3 (da) 2019-01-21
WO2014089290A1 (en) 2014-06-12
US20160298132A1 (en) 2016-10-13
SG10201910987SA (en) 2020-01-30
JP2019037231A (ja) 2019-03-14
US20160017366A1 (en) 2016-01-21
JP2017192392A (ja) 2017-10-26
PL3138911T3 (pl) 2019-04-30
JP2022115994A (ja) 2022-08-09
SG11201503824SA (en) 2015-06-29
US10731181B2 (en) 2020-08-04
CA2891347A1 (en) 2014-06-12
IL238856B (en) 2018-03-29
CN108913676A (zh) 2018-11-30
US20210388396A1 (en) 2021-12-16
PT3360964T (pt) 2019-10-29
JP6620018B2 (ja) 2019-12-11
CN105142669A (zh) 2015-12-09
EP3138909A1 (en) 2017-03-08
IL267598B (en) 2022-05-01
ES2757808T3 (es) 2020-04-30
LT3138911T (lt) 2019-02-25
US20200140897A1 (en) 2020-05-07
KR20200098727A (ko) 2020-08-20
SG10202107423UA (en) 2021-08-30
LT3363902T (lt) 2020-02-10
KR20190093680A (ko) 2019-08-09
EP3363902A1 (en) 2018-08-22
ES2769310T3 (es) 2020-06-25
EP2928496A4 (en) 2017-03-01
US20160298138A1 (en) 2016-10-13
DK3360964T3 (da) 2019-10-28
IL291129B2 (en) 2023-07-01
IL300199A (en) 2023-03-01
AU2022200330A1 (en) 2022-02-17
EP2928496B1 (en) 2019-10-09
AU2023216829B2 (en) 2025-10-09
ES2713243T3 (es) 2019-05-20
EP2928496A1 (en) 2015-10-14
IL267598A (en) 2019-08-29
JP7478772B2 (ja) 2024-05-07
DK2928496T3 (da) 2019-11-11
EP3611263A1 (en) 2020-02-19
AU2017204031B2 (en) 2018-06-14
IL300199B2 (en) 2025-07-01
EP3138910B1 (en) 2017-09-20
KR102531576B1 (ko) 2023-05-11
PL2928496T3 (pl) 2020-04-30
US20160298136A1 (en) 2016-10-13
AU2017204031A1 (en) 2017-07-06
PL3138910T3 (pl) 2018-01-31
AU2019201344A1 (en) 2019-03-21
PT3138910T (pt) 2017-10-18
AU2020230246B2 (en) 2020-11-05
US20160298135A1 (en) 2016-10-13
EP3360964B1 (en) 2019-10-02
LT3138912T (lt) 2019-02-25
IL291129A (en) 2022-05-01
US20160298133A1 (en) 2016-10-13
JP2020120674A (ja) 2020-08-13
PT3138912T (pt) 2018-12-28
AU2013355214A1 (en) 2015-06-04
KR20230003624A (ko) 2023-01-06

Similar Documents

Publication Publication Date Title
AU2022200330B2 (en) Crispr-based genome modification and regulation