[go: up one dir, main page]

PL3867372T3 - Sposoby ukierunkowanego wprowadzania dna do genów - Google Patents

Sposoby ukierunkowanego wprowadzania dna do genów

Info

Publication number
PL3867372T3
PL3867372T3 PL19795751.7T PL19795751T PL3867372T3 PL 3867372 T3 PL3867372 T3 PL 3867372T3 PL 19795751 T PL19795751 T PL 19795751T PL 3867372 T3 PL3867372 T3 PL 3867372T3
Authority
PL
Poland
Prior art keywords
genes
dna
methods
targeted insertion
targeted
Prior art date
Application number
PL19795751.7T
Other languages
English (en)
Inventor
Nicholas BALTES
Original Assignee
Blueallele Corporation
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=68393107&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=PL3867372(T3) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Blueallele Corporation filed Critical Blueallele Corporation
Publication of PL3867372T3 publication Critical patent/PL3867372T3/pl

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/10Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102Mutagenizing nucleic acids
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • C12N15/864Parvoviral vectors, e.g. parvovirus, densovirus
    • C12N15/8645Adeno-associated virus
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90Stable introduction of foreign DNA into chromosome
    • C12N15/902Stable introduction of foreign DNA into chromosome using homologous recombination
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90Stable introduction of foreign DNA into chromosome
    • C12N15/902Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/16Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/20Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/34Allele or polymorphism specific uses
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2800/00Nucleic acids vectors
    • C12N2800/80Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2840/00Vectors comprising a special translation-regulating system
    • C12N2840/44Vectors comprising a special translation-regulating system being a specific part of the splice mechanism, e.g. donor, acceptor

Landscapes

  • Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Mycology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Virology (AREA)
  • Cell Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Peptides Or Proteins (AREA)
PL19795751.7T 2018-10-16 2019-10-14 Sposoby ukierunkowanego wprowadzania dna do genów PL3867372T3 (pl)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201862746497P 2018-10-16 2018-10-16
US201962830654P 2019-04-08 2019-04-08
US201962864432P 2019-06-20 2019-06-20
PCT/US2019/056083 WO2020081438A1 (en) 2018-10-16 2019-10-14 Methods for targeted insertion of dna in genes

Publications (1)

Publication Number Publication Date
PL3867372T3 true PL3867372T3 (pl) 2025-06-23

Family

ID=68393107

Family Applications (1)

Application Number Title Priority Date Filing Date
PL19795751.7T PL3867372T3 (pl) 2018-10-16 2019-10-14 Sposoby ukierunkowanego wprowadzania dna do genów

Country Status (18)

Country Link
US (12) US20200115700A1 (pl)
EP (2) EP4585692A3 (pl)
JP (1) JP7460643B2 (pl)
KR (1) KR20210091167A (pl)
CN (1) CN113166754A (pl)
AU (2) AU2019360182B2 (pl)
BR (1) BR112021007229A2 (pl)
CA (1) CA3116553A1 (pl)
DE (1) DE112019005166B4 (pl)
ES (1) ES3023563T3 (pl)
GB (2) GB2611929B (pl)
HR (1) HRP20250570T1 (pl)
HU (1) HUE071002T2 (pl)
IL (1) IL282290A (pl)
NZ (1) NZ775055A (pl)
PL (1) PL3867372T3 (pl)
SG (1) SG11202103917VA (pl)
WO (1) WO2020081438A1 (pl)

Families Citing this family (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2019090174A1 (en) 2017-11-02 2019-05-09 Arbor Biotechnologies, Inc. Novel crispr-associated transposon systems and components
GB2611929B (en) 2018-10-16 2023-11-22 Blueallele Corp Methods for targeted insertion of DNA in genes
EP3867381A2 (en) * 2018-10-18 2021-08-25 Intellia Therapeutics, Inc. Compositions and methods for transgene expression from an albumin locus
CN113272428A (zh) * 2018-10-18 2021-08-17 英特利亚治疗股份有限公司 核酸构建体和使用方法
CN113195721A (zh) * 2018-10-18 2021-07-30 英特利亚治疗股份有限公司 治疗α-1抗胰蛋白酶缺乏症的组合物和方法
BR112021007301A2 (pt) * 2018-10-18 2021-07-27 Intellia Therapeutics, Inc. composições e métodos para expressar fator ix
EP4054651A1 (en) * 2019-11-08 2022-09-14 Regeneron Pharmaceuticals, Inc. Crispr and aav strategies for x-linked juvenile retinoschisis therapy
WO2021202421A1 (en) * 2020-03-30 2021-10-07 Blueallele, Llc Methods for integrating dna into genes with gain-of-function or loss-of-function mutations
PE20231570A1 (es) * 2020-09-29 2023-10-04 Neuexcell Therapeutics Inc Vector neurod1
US20240018493A1 (en) * 2020-11-10 2024-01-18 The Board Of Trustees Of The Leland Stanford Junior University Knock-in of large dna for long-term high genomic expression
WO2025074350A2 (en) * 2023-10-06 2025-04-10 Universidade De Coimbra Crispr-cas9 based tools: potential novel therapeutic approaches for machado-joseph disease
CN119552872B (zh) * 2025-02-06 2025-04-22 中国科学院天津工业生物技术研究所 一种人工合成启动子Pw及其在产维生素B12菌株中的应用
CN119570796B (zh) * 2025-02-06 2025-04-22 中国科学院天津工业生物技术研究所 一种人工合成启动子ph及其在产维生素b12菌株中的应用

Family Cites Families (97)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DK0695361T4 (da) 1993-04-21 2010-03-22 Univ Edinburgh Ekspression af heterologe gener ifølge en målekspressionsprofil
CA2276529A1 (en) 1996-12-31 1998-07-09 George A. Gaitanaris Vectors and methods for the mutagenesis of mammalian genes
US6740503B1 (en) * 1997-09-26 2004-05-25 Athersys, Inc. Compositions and methods for non-targeted activation of endogenous genes
WO2003087341A2 (en) * 2002-01-23 2003-10-23 The University Of Utah Research Foundation Targeted chromosomal mutagenesis using zinc finger nucleases
US20040106566A1 (en) 2002-05-17 2004-06-03 Shi-Lung Lin RNA-splicing and processing-directed gene silencing and the relative applications thereof
US7888121B2 (en) * 2003-08-08 2011-02-15 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
US20140271782A1 (en) 2013-03-15 2014-09-18 Dermazone Solutions, Inc. Method for preparing nanolipids with encapsulated alcohol
GB0903207D0 (en) * 2009-02-25 2009-04-08 Ucb Pharma Sa Method for expressing multimeric proteins
HUE044522T2 (hu) 2009-04-30 2019-10-28 Univ Pennsylvania Adeno-asszociált vírus konstrukciókat tartalmazó légúti vezetõ sejtek célzására szolgáló kompozíciók
WO2011086118A1 (en) 2010-01-14 2011-07-21 Deutsches Krebsforschungszentrum In vivo determination of the lokalization of dna double-stand breaks and application thereof
WO2012051343A1 (en) 2010-10-12 2012-04-19 The Children's Hospital Of Philadelphia Methods and compositions for treating hemophilia b
CA3177512A1 (en) * 2011-07-15 2013-01-24 E.I. Du Pont De Nemours And Company Novel plant terminator sequences
CA3186126A1 (en) 2011-09-21 2013-03-28 Sangamo Biosciences, Inc. Methods and compositions for regulation of transgene expression
WO2013075008A1 (en) 2011-11-16 2013-05-23 University Of Florida Research Foundation Inc. Aav dual vector systems for gene therapy
AU2013232114B2 (en) 2012-03-15 2018-10-18 Omeros Corporation Composition and method for diversification of target sequences
ES2828663T3 (es) 2012-04-18 2021-05-27 Univ Leland Stanford Junior Dirección genética no disruptiva
BR112014027813A2 (pt) 2012-05-07 2017-08-08 Dow Agrosciences Llc métodos e composições para integração de transgenes direcionada mediada por nuclease
US10648001B2 (en) 2012-07-11 2020-05-12 Sangamo Therapeutics, Inc. Method of treating mucopolysaccharidosis type I or II
ES2697912T3 (es) 2012-07-11 2019-01-29 Sangamo Therapeutics Inc Métodos y composiciones para el tratamiento de enfermedades monogénicas
UA119135C2 (uk) 2012-09-07 2019-05-10 ДАУ АГРОСАЙЄНСІЗ ЕлЕлСі Спосіб отримання трансгенної рослини
AU2013355327A1 (en) 2012-12-05 2015-06-11 Sangamo Therapeutics, Inc. Methods and compositions for regulation of metabolic disorders
PT3363902T (pt) 2012-12-06 2019-12-19 Sigma Aldrich Co Llc Modificação e regulação de genoma baseadas em crispr
US20140273235A1 (en) 2013-03-15 2014-09-18 Regents Of The University Of Minnesota ENGINEERING PLANT GENOMES USING CRISPR/Cas SYSTEMS
WO2014172458A1 (en) 2013-04-16 2014-10-23 University Of Washington Through Its Center For Commercialization Activating an alternative pathway for homology-directed repair to stimulate targeted gene correction and genome engineering
AU2014265331B2 (en) 2013-05-15 2019-12-05 Sangamo Therapeutics, Inc. Methods and compositions for treatment of a genetic condition
CA2920253A1 (en) 2013-08-02 2015-02-05 Enevolv, Inc. Processes and host cells for genome, pathway, and biomolecular engineering
AU2014315335B2 (en) 2013-09-04 2017-08-24 Corteva Agriscience Llc Rapid targeting analysis in crops for determining donor insertion
AU2014341928B2 (en) 2013-11-04 2017-11-30 Corteva Agriscience Llc A universal donor system for gene targeting
US20160298096A1 (en) 2013-11-18 2016-10-13 Crispr Therapeutics Ag Crispr-cas system materials and methods
EP3757116A1 (en) 2013-12-09 2020-12-30 Sangamo Therapeutics, Inc. Methods and compositions for genome engineering
SG10201804973TA (en) * 2013-12-12 2018-07-30 Broad Inst Inc Compositions and Methods of Use of Crispr-Cas Systems in Nucleotide Repeat Disorders
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
WO2015134812A1 (en) 2014-03-05 2015-09-11 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating usher syndrome and retinitis pigmentosa
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)
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
EP3556858A3 (en) 2014-04-09 2020-01-22 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating cystic fibrosis
GB201407852D0 (en) 2014-05-02 2014-06-18 Iontas Ltd Preparation of libraries od protein variants expressed in eukaryotic cells and use for selecting binding molecules
WO2015173436A1 (en) 2014-05-16 2015-11-19 Vrije Universiteit Brussel Genetic correction of myotonic dystrophy type 1
US9970030B2 (en) 2014-08-27 2018-05-15 Caribou Biosciences, Inc. Methods for increasing CAS9-mediated engineering efficiency
EP3204513A2 (en) 2014-10-09 2017-08-16 Life Technologies Corporation Crispr oligonucleotides and gene editing
AU2015342749B2 (en) 2014-11-07 2022-01-27 Editas Medicine, Inc. Methods for improving CRISPR/Cas-mediated genome-editing
US11427829B2 (en) 2014-12-16 2022-08-30 Danisco Us Inc Fungal genome modification systems and methods of use
CA2972454C (en) * 2014-12-31 2024-09-10 Synthetic Genomics Inc Compositions and methods for high efficiency in vivo genome editing
JP6879486B2 (ja) 2015-03-17 2021-06-02 フリーイェ・ユニヴェルシテイト・ブリュッセルVrije Universieit Brussel Fviiiおよびfix用の最適化された肝臓特異的発現系
WO2016154596A1 (en) 2015-03-25 2016-09-29 Editas Medicine, Inc. Crispr/cas-related methods, compositions and components
EP3274453B1 (en) 2015-03-26 2021-01-27 Editas Medicine, Inc. Crispr/cas-mediated gene conversion
EP3748004A1 (en) 2015-04-01 2020-12-09 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating duchenne muscular dystrophy and becker muscular dystrophy
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
WO2016176191A1 (en) 2015-04-27 2016-11-03 The Trustees Of The University Of Pennsylvania Dual aav vector system for crispr/cas9 mediated correction of human disease
EP3294888A1 (en) 2015-05-11 2018-03-21 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating hiv infection and aids
EP3294896A1 (en) 2015-05-11 2018-03-21 Editas Medicine, Inc. Optimized crispr/cas9 systems and methods for gene editing in stem cells
WO2016201047A1 (en) 2015-06-09 2016-12-15 Editas Medicine, Inc. Crispr/cas-related methods and compositions for improving transplantation
WO2016205680A1 (en) 2015-06-17 2016-12-22 The Uab Research Foundation Crispr/cas9 complex for introducing a functional polypeptide into cells of blood cell lineage
US10526590B2 (en) 2015-08-31 2020-01-07 Agilent Technologies, Inc. Compounds and methods for CRISPR/Cas-based genome editing by homologous recombination
WO2017048995A1 (en) 2015-09-15 2017-03-23 Mirimus, Inc. Inducible crispr/cas9 and rnai systems and methods of use
CA2999500A1 (en) 2015-09-24 2017-03-30 Editas Medicine, Inc. Use of exonucleases to improve crispr/cas-mediated genome editing
EP3350327B1 (en) 2015-10-23 2018-09-26 Caribou Biosciences, Inc. Engineered crispr class 2 cross-type nucleic-acid targeting nucleic acids
MY189674A (en) * 2015-10-28 2022-02-24 Sangamo Therapeutics Inc Liver-specific constructs, factor viii expression cassettes and methods of use thereof
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
EP3402533B1 (en) 2016-01-15 2021-08-04 Sangamo Therapeutics, Inc. Methods and compositions for the treatment of neurologic disease
US20200291369A1 (en) 2016-03-11 2020-09-17 Erasmus University Medical Center Rotterdam Improved CRISPR-Cas9 Genome Editing Tool
US20200291368A1 (en) 2016-03-11 2020-09-17 Wageningen Universiteit Improved CRISPR-Cpf1 Genome Editing Tool
WO2017158422A1 (en) 2016-03-16 2017-09-21 Crispr Therapeutics Ag Materials and methods for treatment of hereditary haemochromatosis
EP4047092B1 (en) 2016-04-13 2025-07-30 Editas Medicine, Inc. Cas9 fusion molecules, gene editing systems, and methods of use thereof
AU2017248659B2 (en) 2016-04-15 2022-08-11 The Trustees Of The University Of Pennsylvania Gene therapy for treating hemophilia A
ES2957660T3 (es) 2016-05-05 2024-01-23 Univ Duke Composiciones relacionadas con crispr/cas para tratar la distrofia muscular de duchenne
WO2017205650A1 (en) 2016-05-27 2017-11-30 Life Technologies Corporation Compositions and methods for enhancing homologous recombination
WO2017216771A2 (en) 2016-06-17 2017-12-21 Genesis Technologies Limited Crispr-cas system, materials and methods
MX2019000262A (es) 2016-07-05 2019-05-27 Univ Johns Hopkins Composiciones basadas en crispr/cas9 y metodos para el tratamiento de degeneraciones retinianas.
EP3481956A4 (en) * 2016-07-05 2020-07-08 The Johns Hopkins University COMPOSITIONS AND METHODS COMPRISING CRISPR-GUIDED RNA ENHANCEMENTS USING THE H1 PROMOTER
JP6829761B2 (ja) 2016-09-29 2021-02-10 エフ.ホフマン−ラ ロシュ アーゲーF. Hoffmann−La Roche Aktiengesellschaft 遺伝子編集モジュールおよび遺伝子送達アプローチを解析および最適化するための方法
US20230190958A1 (en) * 2017-01-13 2023-06-22 Jichi Medical University AAV Vector for Disrupting Coagulation Factor-Related Gene on Liver Genome
AU2018221730B2 (en) 2017-02-15 2024-06-20 Novo Nordisk A/S Donor repair templates multiplex genome editing
WO2018191715A2 (en) 2017-04-14 2018-10-18 Synthetic Genomics, Inc. Polypeptides with type v crispr activity and uses thereof
WO2018195555A1 (en) 2017-04-21 2018-10-25 The Board Of Trustees Of The Leland Stanford Junior University Crispr/cas 9-mediated integration of polynucleotides by sequential homologous recombination of aav donor vectors
WO2018197020A1 (en) 2017-04-27 2018-11-01 Novozymes A/S Genome editing by crispr-cas9 using short donor oligonucleotides
US20200123567A1 (en) 2017-06-26 2020-04-23 Samira Kiani A universal platform to enhance crispr-based gene editing for in vivo therapies
CN111315883B (zh) * 2017-07-18 2024-06-18 杰诺维有限公司 用于全长t细胞受体可读框的快速装配和多样化的两组分载体文库系统
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
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
WO2019079763A1 (en) 2017-10-19 2019-04-25 Cola Gary M Jr HIGH STRENGTH STEELS, METHODS OF MANUFACTURE THEREOF AND ARTICLES RESULTING FROM THE SAME
WO2019092507A2 (en) 2017-11-09 2019-05-16 Crispr Therapeutics Ag Crispr/cas systems for treatment of dmd
BR112020011255A2 (pt) 2017-12-05 2020-11-24 Vertex Pharmaceuticals Incorporated células-tronco e progenitoras hematopoiéticas cd34+ humanas modificadas por crispr-cas9 e usos das mesmas
WO2019118875A1 (en) 2017-12-15 2019-06-20 Ou Li Crispr-mediated genome editing with vectors
WO2019157326A1 (en) 2018-02-08 2019-08-15 Zymergen Inc. Genome editing using crispr in corynebacterium
JP7611564B2 (ja) 2018-03-13 2025-01-10 リージェンツ オブ ザ ユニバーシティ オブ ミネソタ Cas9塩基エディターを使用するリンパ球造血系操作
CN117757842A (zh) 2018-03-15 2024-03-26 星际治疗有限公司 合成dna运载体及使用方法
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
JP2021521838A (ja) 2018-04-27 2021-08-30 シアトル チルドレンズ ホスピタル (ディービーエイ シアトル チルドレンズ リサーチ インスティテュート) ブルトン型チロシンキナーゼに対するtalenベースのおよびcrispr/casベースのゲノム編集
US11827877B2 (en) * 2018-06-28 2023-11-28 Crispr Therapeutics Ag Compositions and methods for genomic editing by insertion of donor polynucleotides
US20210317436A1 (en) 2018-09-08 2021-10-14 Blueallele, Llc Methods and compositions for modifying the von willebrand factor gene
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
CN113195721A (zh) 2018-10-18 2021-07-30 英特利亚治疗股份有限公司 治疗α-1抗胰蛋白酶缺乏症的组合物和方法
EP3867381A2 (en) 2018-10-18 2021-08-25 Intellia Therapeutics, Inc. Compositions and methods for transgene expression from an albumin locus
CN113272428A (zh) 2018-10-18 2021-08-17 英特利亚治疗股份有限公司 核酸构建体和使用方法

Also Published As

Publication number Publication date
GB2593353A (en) 2021-09-22
EP3867372C0 (en) 2025-04-02
ES3023563T3 (en) 2025-06-02
IL282290A (en) 2021-05-31
US11091756B2 (en) 2021-08-17
AU2019360182B2 (en) 2023-02-16
JP7460643B2 (ja) 2024-04-02
US20240141322A1 (en) 2024-05-02
US20240141323A1 (en) 2024-05-02
US20200115700A1 (en) 2020-04-16
EP4585692A3 (en) 2025-09-17
US20220154168A1 (en) 2022-05-19
KR20210091167A (ko) 2021-07-21
US20210332347A1 (en) 2021-10-28
JP2022513365A (ja) 2022-02-07
DE112019005166T5 (de) 2021-07-29
GB202300487D0 (en) 2023-03-01
US11254930B2 (en) 2022-02-22
AU2019360182A1 (en) 2021-05-20
EP3867372B8 (en) 2025-05-07
US20200190504A1 (en) 2020-06-18
HRP20250570T1 (hr) 2025-07-04
US12054706B2 (en) 2024-08-06
CA3116553A1 (en) 2020-04-23
BR112021007229A2 (pt) 2021-08-10
CN113166754A (zh) 2021-07-23
DE112019005166B4 (de) 2024-12-24
US20240141320A1 (en) 2024-05-02
NZ775055A (en) 2024-11-29
US20240141319A1 (en) 2024-05-02
US12441994B2 (en) 2025-10-14
GB2593353B (en) 2023-03-01
EP3867372A1 (en) 2021-08-25
US11365407B2 (en) 2022-06-21
HUE071002T2 (hu) 2025-07-28
GB2611929B (en) 2023-11-22
US20240141318A1 (en) 2024-05-02
GB202106906D0 (en) 2021-06-30
EP4585692A2 (en) 2025-07-16
SG11202103917VA (en) 2021-05-28
US20230212553A1 (en) 2023-07-06
WO2020081438A1 (en) 2020-04-23
US11993770B2 (en) 2024-05-28
AU2023202878A1 (en) 2023-05-25
GB2611929A (en) 2023-04-19
US20240141317A1 (en) 2024-05-02
EP3867372B1 (en) 2025-04-02
US20240141321A1 (en) 2024-05-02

Similar Documents

Publication Publication Date Title
IL282290A (en) Methods for introducing DNA into genes
IL283585B1 (en) Methods and preparations for targeted genetic modification using paired guide RNA
IL288263A (en) CRISPR DNA/RNA hybrid polynucleotides and methods for using them
IL265953A (en) Gene insertion is targeted for improved immune cell therapy
IL256905A (en) Methods for Amplifying Nucleic Acid Sequences
EP3245220A4 (en) Methods and compositions for targeted gene transfer
IL257524B1 (en) Short interfering RNA and its use in methods and preparations for inhibiting the expression of ankyrin horseradish protein gene regulated by Nutz
HUE061474T2 (hu) Nukleinsav-szekvenálási eljárás
SG11202106810UA (en) Methods for targeted complementary dna enrichment
HK40007544A (en) Targeted gene insertion for improved immune cells therapy