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MX2023004383A - Sistemas, metodos y composiciones para la ingenieria genetica especifica del sitio usando la adicion programable a traves de elementos objetivo especificos del sitio (paste). - Google Patents

Sistemas, metodos y composiciones para la ingenieria genetica especifica del sitio usando la adicion programable a traves de elementos objetivo especificos del sitio (paste).

Info

Publication number
MX2023004383A
MX2023004383A MX2023004383A MX2023004383A MX2023004383A MX 2023004383 A MX2023004383 A MX 2023004383A MX 2023004383 A MX2023004383 A MX 2023004383A MX 2023004383 A MX2023004383 A MX 2023004383A MX 2023004383 A MX2023004383 A MX 2023004383A
Authority
MX
Mexico
Prior art keywords
site
paste
specific
compositions
methods
Prior art date
Application number
MX2023004383A
Other languages
English (en)
Inventor
Jonathan Gootenberg
Omar Abudayyeh
Original Assignee
Massachusetts Inst Technology
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
Application filed by Massachusetts Inst Technology filed Critical Massachusetts Inst Technology
Publication of MX2023004383A publication Critical patent/MX2023004383A/es

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    • 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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
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    • 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
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/10—Transferases (2.)
    • C12N9/12—Transferases (2.) transferring phosphorus containing groups, e.g. kinases (2.7)
    • C12N9/1241—Nucleotidyltransferases (2.7.7)
    • C12N9/1276—RNA-directed DNA polymerase (2.7.7.49), i.e. reverse transcriptase or telomerase
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
    • C12Y—ENZYMES
    • C12Y207/00—Transferases transferring phosphorus-containing groups (2.7)
    • C12Y207/07—Nucleotidyltransferases (2.7.7)
    • C12Y207/07049—RNA-directed DNA polymerase (2.7.7.49), i.e. telomerase or reverse-transcriptase
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3519—Fusion with another nucleic acid
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/10011—Adenoviridae
    • C12N2710/10311—Mastadenovirus, e.g. human or simian adenoviruses
    • C12N2710/10341—Use of virus, viral particle or viral elements as a vector
    • C12N2710/10343—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Medicinal Chemistry (AREA)
  • Cell Biology (AREA)
  • Mycology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Epidemiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)

Abstract

La presente descripción proporciona sistemas, métodos y composiciones para la ingeniería genética específica del sitio usando la adición programable a través de elementos objetivo específicos del sitio (PASTE). PASTE comprende la adición de un sitio de integración en un genoma objetivo seguida por la inserción de uno o más genes de interés o una o más secuencias de ácidos nucleicos de interés en el sitio. PASTE combina las tecnologías de edición de genes y las tecnologías de integrasa para lograr la incorporación unidireccional de genes en un genoma para el tratamiento y diagnóstico de enfermedades.
MX2023004383A 2020-10-21 2021-10-21 Sistemas, metodos y composiciones para la ingenieria genetica especifica del sitio usando la adicion programable a traves de elementos objetivo especificos del sitio (paste). MX2023004383A (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063094803P 2020-10-21 2020-10-21
US202163222550P 2021-07-16 2021-07-16
PCT/US2021/056006 WO2022087235A1 (en) 2020-10-21 2021-10-21 Systems, methods, and compositions for site-specific genetic engineering using programmable addition via site-specific targeting elements (paste)

Publications (1)

Publication Number Publication Date
MX2023004383A true MX2023004383A (es) 2023-05-04

Family

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MX2023004383A MX2023004383A (es) 2020-10-21 2021-10-21 Sistemas, metodos y composiciones para la ingenieria genetica especifica del sitio usando la adicion programable a traves de elementos objetivo especificos del sitio (paste).

Country Status (10)

Country Link
US (7) US20220145293A1 (es)
EP (1) EP4232583A1 (es)
JP (1) JP2023546597A (es)
KR (1) KR20230091894A (es)
CN (1) CN116419975A (es)
AU (1) AU2021364781B2 (es)
CA (1) CA3196116A1 (es)
IL (1) IL301368A (es)
MX (1) MX2023004383A (es)
WO (1) WO2022087235A1 (es)

Families Citing this family (40)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20190127797A (ko) 2017-03-10 2019-11-13 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 시토신에서 구아닌으로의 염기 편집제
US12281338B2 (en) 2018-10-29 2025-04-22 The Broad Institute, Inc. Nucleobase editors comprising GeoCas9 and uses thereof
US20220259617A1 (en) 2019-06-13 2022-08-18 The General Hospital Corporation Engineered human-endogenous virus-like particles and methods of use thereof for delivery to cells
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
BR112023001272A2 (pt) 2020-07-24 2023-04-04 Massachusetts Gen Hospital Partículas semelhantes a vírus aprimoradas e métodos de uso das mesmas para entrega às células
IL301368A (en) 2020-10-21 2023-05-01 Massachusetts Inst Technology Systems, Methods, and Compositions for Site-Specific Genetic Engineering Using Programmable Insertion Using Site-Specific Targeting Elements (PASTE)
GB202113933D0 (en) * 2021-09-29 2021-11-10 Genome Res Ltd Methods for gene editing
US20240417719A1 (en) * 2021-10-25 2024-12-19 The Broad Institute, Inc. Methods and compositions for editing a genome with prime editing and a recombinase
WO2023077148A1 (en) * 2021-11-01 2023-05-04 Tome Biosciences, Inc. Single construct platform for simultaneous delivery of gene editing machinery and nucleic acid cargo
WO2023114992A1 (en) * 2021-12-17 2023-06-22 Massachusetts Institute Of Technology Programmable insertion approaches via reverse transcriptase recruitment
CA3244180A1 (en) 2021-12-22 2023-06-29 Tome Biosciences, Inc. CO-ADMINISTRATION OF A GENE EDITOR CONSTRUCTION AND A PATTERN DONOR
EP4511486A1 (en) 2022-04-20 2025-02-26 Massachusetts Institute Of Technology Site specific genetic engineering utilizing trans-template rnas
WO2023205744A1 (en) * 2022-04-20 2023-10-26 Tome Biosciences, Inc. Programmable gene insertion compositions
US20230407280A1 (en) 2022-04-20 2023-12-21 Massachusetts Institute Of Technology Programmable gene editing using guide rna pair
WO2023215831A1 (en) * 2022-05-04 2023-11-09 Tome Biosciences, Inc. Guide rna compositions for programmable gene insertion
WO2023225670A2 (en) * 2022-05-20 2023-11-23 Tome Biosciences, Inc. Ex vivo programmable gene insertion
MA71557A (fr) 2022-07-18 2025-05-30 Renagade Therapeutics Management Inc. Composants d'édition génique, systèmes et procédés d'utilisation
WO2024020587A2 (en) * 2022-07-22 2024-01-25 Tome Biosciences, Inc. Pleiopluripotent stem cell programmable gene insertion
EP4615960A1 (en) 2022-11-09 2025-09-17 C3S2 GmbH Methods for manufacturing engineered immune cells
CN116004586B (zh) * 2022-12-01 2025-08-26 上海药明生物医药有限公司 通过优化提高Bxb1酶的重组效率的方法
WO2024138194A1 (en) 2022-12-22 2024-06-27 Tome Biosciences, Inc. Platforms, compositions, and methods for in vivo programmable gene insertion
KR20250155074A (ko) * 2023-01-05 2025-10-29 프라임 메디슨, 아이엔씨. 게놈 편집 조성물 및 사용 방법
EP4662306A2 (en) * 2023-02-09 2025-12-17 The Broad Institute Inc. Evolved recombinases for editing a genome in combination with prime editing
WO2024220407A2 (en) * 2023-04-17 2024-10-24 Massachusetts Institute Of Technology Systems, methods, and compositions for cystic fibrosis gene editing
WO2024234006A1 (en) 2023-05-11 2024-11-14 Tome Biosciences, Inc. Systems, compositions, and methods for targeting liver sinusodial endothelial cells (lsecs)
WO2024238595A2 (en) * 2023-05-16 2024-11-21 Sarepta Therapeutics, Inc. Fusion proteins comprising cas enzymes and dna-binding proteins
AU2024274412A1 (en) * 2023-05-16 2025-12-11 Prime Medicine, Inc. Genome editing compositions targeting the b2m gene and methods of use
WO2025006468A2 (en) 2023-06-26 2025-01-02 University Of Hawaii Evolved integrases and methods of using the same for genome editing
WO2025049959A2 (en) 2023-09-01 2025-03-06 Renagade Therapeutics Management Inc. Gene editing systems, compositions, and methods for treatment of vexas syndrome
WO2025050069A1 (en) 2023-09-01 2025-03-06 Tome Biosciences, Inc. Programmable gene insertion using engineered integration enzymes
WO2025117544A1 (en) 2023-11-29 2025-06-05 The Broad Institute, Inc. Engineered omega guide molecule and iscb compositions, systems, and methods of use thereof
WO2025128871A2 (en) 2023-12-13 2025-06-19 Renagade Therapeutics Management Inc. Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents
WO2025147545A1 (en) 2024-01-03 2025-07-10 Juno Therapeutics, Inc. Lipid nanoparticles for delivery of nucleic acids and related methods and uses
WO2025155753A2 (en) 2024-01-17 2025-07-24 Renagade Therapeutics Management Inc. Improved gene editing system, guides, and methods
CN117720672B (zh) * 2024-02-07 2024-04-30 深锐(天津)生物医学有限公司 先导编辑系统及其应用
WO2025174765A1 (en) 2024-02-12 2025-08-21 Renagade Therapeutics Management Inc. Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents
WO2025193915A1 (en) 2024-03-14 2025-09-18 Massachusetts Institute Of Technology High efficiency integrases for gene editing
WO2025224107A1 (en) 2024-04-22 2025-10-30 Basecamp Research Ltd Method and compositions for detecting off-target editing
WO2025226730A1 (en) * 2024-04-22 2025-10-30 Transit Therapeutics, Inc. Revision of genetic material using direct replacement 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

Family Cites Families (32)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US8889394B2 (en) 2009-09-07 2014-11-18 Empire Technology Development Llc Multiple domain proteins
WO2013176772A1 (en) 2012-05-25 2013-11-28 The Regents Of The University Of California Methods and compositions for rna-directed target dna modification and for rna-directed modulation of transcription
WO2014093655A2 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Engineering and optimization of systems, methods and compositions for sequence manipulation with functional domains
SG10201704932UA (en) 2012-12-17 2017-07-28 Harvard College Rna-guided human genome engineering
NZ712727A (en) 2013-03-14 2017-05-26 Caribou Biosciences Inc Compositions and methods of nucleic acid-targeting nucleic acids
US20140349400A1 (en) * 2013-03-15 2014-11-27 Massachusetts Institute Of Technology Programmable Modification of DNA
US10563225B2 (en) 2013-07-26 2020-02-18 President And Fellows Of Harvard College Genome engineering
US9228207B2 (en) 2013-09-06 2016-01-05 President And Fellows Of Harvard College Switchable gRNAs comprising aptamers
US9322037B2 (en) * 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
AU2015277180A1 (en) 2014-06-17 2017-01-12 Poseida Therapeutics, Inc. A method for directing proteins to specific loci in the genome and uses thereof
US10059940B2 (en) 2015-01-27 2018-08-28 Minghong Zhong Chemically ligated RNAs for CRISPR/Cas9-lgRNA complexes as antiviral therapeutic agents
WO2016205728A1 (en) 2015-06-17 2016-12-22 Massachusetts Institute Of Technology Crispr mediated recording of cellular events
US11447768B2 (en) 2016-03-01 2022-09-20 University Of Florida Research Foundation, Incorporated Molecular cell diary system
EP3443088B1 (en) 2016-04-13 2024-09-18 Editas Medicine, Inc. Grna fusion molecules, gene editing systems, and methods of use thereof
KR102827276B1 (ko) 2016-08-03 2025-07-01 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 아데노신 핵염기 편집제 및 그의 용도
ES2985812T3 (es) 2016-09-09 2024-11-07 The Board Of Trustees Of The Leland Stanfordjunior Univ Edición genómica de precisión de alto rendimiento
US11279612B2 (en) 2016-09-12 2022-03-22 MEMS Drive (Nanjing) Co., Ltd. MEMS actuation systems and methods
KR20190127797A (ko) 2017-03-10 2019-11-13 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 시토신에서 구아닌으로의 염기 편집제
WO2019051097A1 (en) 2017-09-08 2019-03-14 The Regents Of The University Of California RNA-GUIDED ENDONUCLEASE FUSION POLYPEPTIDES AND METHODS OF USING SAME
EP3724332A1 (en) 2017-12-14 2020-10-21 CRISPR Therapeutics AG Novel rna-programmable endonuclease systems and their use in genome editing and other applications
WO2020047124A1 (en) 2018-08-28 2020-03-05 Flagship Pioneering, Inc. Methods and compositions for modulating a genome
DE112020001306T5 (de) 2019-03-19 2022-01-27 Massachusetts Institute Of Technology Verfahren und zusammensetzungen zur editierung von nukleotidsequenzen
EP3953477A4 (en) 2019-06-06 2022-06-22 Inscripta, Inc. HARDENING FOR RECURSIVE NUCLEIC ACID-DRIVEN CELL EDITING
AU2020341479A1 (en) 2019-09-03 2022-03-31 Myeloid Therapeutics, Inc. Methods and compositions for genomic integration
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
EP4085141A4 (en) * 2019-12-30 2024-03-06 The Broad Institute, Inc. GENOME EDITING USING ACTIVATED, FULLY ACTIVE CRISPR COMPLEXES OF REVERSE TRANSCRIPTASE
WO2021188840A1 (en) 2020-03-19 2021-09-23 Rewrite Therapeutics, Inc. Methods and compositions for directed genome editing
AU2021267940A1 (en) 2020-05-08 2022-12-08 President And Fellows Of Harvard College Methods and compositions for simultaneous editing of both strands of a target double-stranded nucleotide sequence
US11299731B1 (en) * 2020-09-15 2022-04-12 Inscripta, Inc. CRISPR editing to embed nucleic acid landing pads into genomes of live cells
WO2022067130A2 (en) 2020-09-24 2022-03-31 The Broad Institute, Inc. Prime editing guide rnas, compositions thereof, and methods of using the same
IL301368A (en) 2020-10-21 2023-05-01 Massachusetts Inst Technology Systems, Methods, and Compositions for Site-Specific Genetic Engineering Using Programmable Insertion Using Site-Specific Targeting Elements (PASTE)
CN116829706A (zh) 2020-11-05 2023-09-29 华盛顿大学 基于引导编辑的精确的基因组删除和替换方法

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CA3196116A1 (en) 2022-04-28
WO2022087235A1 (en) 2022-04-28
US20220154224A1 (en) 2022-05-19
KR20230091894A (ko) 2023-06-23
US20240067961A1 (en) 2024-02-29
CN116419975A (zh) 2023-07-11
US11572556B2 (en) 2023-02-07
US20240076662A1 (en) 2024-03-07
US12195733B2 (en) 2025-01-14
US11952571B2 (en) 2024-04-09
US11834658B2 (en) 2023-12-05
IL301368A (en) 2023-05-01
US20250092390A1 (en) 2025-03-20
EP4232583A1 (en) 2023-08-30
US20230135673A1 (en) 2023-05-04
US20230279391A1 (en) 2023-09-07
US20220145293A1 (en) 2022-05-12
AU2021364781B2 (en) 2025-10-09
AU2021364781A1 (en) 2023-06-01
JP2023546597A (ja) 2023-11-06
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