JP2021513338A5 - - Google Patents

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Publication number
JP2021513338A5
JP2021513338A5 JP2020542727A JP2020542727A JP2021513338A5 JP 2021513338 A5 JP2021513338 A5 JP 2021513338A5 JP 2020542727 A JP2020542727 A JP 2020542727A JP 2020542727 A JP2020542727 A JP 2020542727A JP 2021513338 A5 JP2021513338 A5 JP 2021513338A5
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subject
sequence
zfn
composition
aav vector
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JP2020542727A
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Japanese (ja)
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JP7275152B2 (ja
JP2021513338A (ja
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Priority claimed from PCT/US2019/017273 external-priority patent/WO2019157324A1/en
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Priority to JP2023076356A priority Critical patent/JP2023090871A/ja
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JP2020542727A 2018-02-08 2019-02-08 操作された標的特異的なヌクレアーゼ Active JP7275152B2 (ja)

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JP2023076356A JP2023090871A (ja) 2018-02-08 2023-05-02 操作された標的特異的なヌクレアーゼ

Applications Claiming Priority (11)

Application Number Priority Date Filing Date Title
US201862628016P 2018-02-08 2018-02-08
US62/628,016 2018-02-08
US201862728226P 2018-09-07 2018-09-07
US62/728,226 2018-09-07
US201862758786P 2018-11-12 2018-11-12
US62/758,786 2018-11-12
US201962795937P 2019-01-23 2019-01-23
US62/795,937 2019-01-23
US201962802092P 2019-02-06 2019-02-06
US62/802,092 2019-02-06
PCT/US2019/017273 WO2019157324A1 (en) 2018-02-08 2019-02-08 Engineered target specific nucleases

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JP2023076356A Division JP2023090871A (ja) 2018-02-08 2023-05-02 操作された標的特異的なヌクレアーゼ

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JP2021513338A JP2021513338A (ja) 2021-05-27
JP2021513338A5 true JP2021513338A5 (enExample) 2022-02-07
JP7275152B2 JP7275152B2 (ja) 2023-05-17

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JP2023076356A Pending JP2023090871A (ja) 2018-02-08 2023-05-02 操作された標的特異的なヌクレアーゼ

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US (2) US11401512B2 (enExample)
EP (1) EP3749350A4 (enExample)
JP (2) JP7275152B2 (enExample)
KR (1) KR20200118468A (enExample)
CN (1) CN111954540A (enExample)
AU (1) AU2019218118A1 (enExample)
BR (1) BR112020015488A2 (enExample)
CA (1) CA3089587A1 (enExample)
IL (1) IL276490A (enExample)
MX (1) MX2020008272A (enExample)
RU (1) RU2020129448A (enExample)
SG (1) SG11202007440YA (enExample)
WO (1) WO2019157324A1 (enExample)

Families Citing this family (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2385251B1 (es) * 2009-05-06 2013-05-06 Fundació Privada Institut D'investigació Biomèdica De Bellvitge Adenovirus oncolíticos para el tratamiento del cáncer.
KR102712926B1 (ko) 2016-10-20 2024-10-07 상가모 테라퓨틱스, 인코포레이티드 파브리병의 치료를 위한 방법 및 조성물
CA3066641A1 (en) * 2017-06-16 2018-12-20 Sangamo Therapeutics, Inc. Targeted disruption of t cell and/or hla receptors
US11512287B2 (en) * 2017-06-16 2022-11-29 Sangamo Therapeutics, Inc. Targeted disruption of T cell and/or HLA receptors
US20200063160A1 (en) * 2018-08-07 2020-02-27 Sangamo Therapeutics, Inc. Method for the treatment of mucopolysaccharidosis type ii
BR112021003399A2 (pt) 2018-08-24 2021-05-18 Spark Therapeutics, Inc. sequências de promotores otimizadas, construtos de expressão livres de íntron e métodos de uso
CN113423419A (zh) * 2019-01-04 2021-09-21 桑格摩生物治疗股份有限公司 用于治疗法布里病的方法和组合物
AU2019428629A1 (en) * 2019-02-06 2021-01-28 Sangamo Therapeutics, Inc. Method for the treatment of mucopolysaccharidosis type I
WO2021087361A1 (en) * 2019-11-01 2021-05-06 Sangamo Therapeutics, Inc. Zinc finger nuclease variants for treating or preventing lysosomal storage diseases
EP4165196A1 (en) * 2020-06-11 2023-04-19 Sangamo Therapeutics, Inc. Methods and compositions for expressing phenylalanine hydroxylase
JP2024515936A (ja) * 2021-03-19 2024-04-11 メタゲノミ,インク. Cas酵素を用いたマルチプレックス編集
CN113293165B (zh) * 2021-06-21 2022-11-15 军事科学院军事医学研究院环境医学与作业医学研究所 一种基于CRISPR-Cas12a技术的HEV特异crRNA、检测试剂盒及其应用
US20230242922A1 (en) * 2021-09-13 2023-08-03 Life Technologies Corporation Gene editing tools

Family Cites Families (83)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5487994A (en) 1992-04-03 1996-01-30 The Johns Hopkins University Insertion and deletion mutants of FokI restriction endonuclease
US5436150A (en) 1992-04-03 1995-07-25 The Johns Hopkins University Functional domains in flavobacterium okeanokoities (foki) restriction endonuclease
US5356802A (en) 1992-04-03 1994-10-18 The Johns Hopkins University Functional domains in flavobacterium okeanokoites (FokI) restriction endonuclease
DE69534629D1 (de) 1994-01-18 2005-12-29 Scripps Research Inst Derivate von zinkfingerproteinen und methoden
US6242568B1 (en) 1994-01-18 2001-06-05 The Scripps Research Institute Zinc finger protein derivatives and methods therefor
US6140466A (en) 1994-01-18 2000-10-31 The Scripps Research Institute Zinc finger protein derivatives and methods therefor
USRE39229E1 (en) 1994-08-20 2006-08-08 Gendaq Limited Binding proteins for recognition of DNA
GB9824544D0 (en) 1998-11-09 1999-01-06 Medical Res Council Screening system
US5789538A (en) 1995-02-03 1998-08-04 Massachusetts Institute Of Technology Zinc finger proteins with high affinity new DNA binding specificities
US5925523A (en) 1996-08-23 1999-07-20 President & Fellows Of Harvard College Intraction trap assay, reagents and uses thereof
GB2338237B (en) 1997-02-18 2001-02-28 Actinova Ltd In vitro peptide or protein expression library
GB9703369D0 (en) 1997-02-18 1997-04-09 Lindqvist Bjorn H Process
GB9710809D0 (en) 1997-05-23 1997-07-23 Medical Res Council Nucleic acid binding proteins
GB9710807D0 (en) 1997-05-23 1997-07-23 Medical Res Council Nucleic acid binding proteins
US6410248B1 (en) 1998-01-30 2002-06-25 Massachusetts Institute Of Technology General strategy for selecting high-affinity zinc finger proteins for diverse DNA target sites
ES2341926T3 (es) 1998-03-02 2010-06-29 Massachusetts Institute Of Technology Poliproteinas con dedos de cinc que tienen enlazadores mejorados.
US6140081A (en) 1998-10-16 2000-10-31 The Scripps Research Institute Zinc finger binding domains for GNN
US6534261B1 (en) 1999-01-12 2003-03-18 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
US7013219B2 (en) 1999-01-12 2006-03-14 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
US6599692B1 (en) 1999-09-14 2003-07-29 Sangamo Bioscience, Inc. Functional genomics using zinc finger proteins
US7070934B2 (en) 1999-01-12 2006-07-04 Sangamo Biosciences, Inc. Ligand-controlled regulation of endogenous gene expression
US6453242B1 (en) 1999-01-12 2002-09-17 Sangamo Biosciences, Inc. Selection of sites for targeting by zinc finger proteins and methods of designing zinc finger proteins to bind to preselected sites
US7030215B2 (en) 1999-03-24 2006-04-18 Sangamo Biosciences, Inc. Position dependent recognition of GNN nucleotide triplets by zinc fingers
US6794136B1 (en) 2000-11-20 2004-09-21 Sangamo Biosciences, Inc. Iterative optimization in the design of binding proteins
US6379903B1 (en) 1999-10-08 2002-04-30 Sigma-Aldrich Co. Purification of recombinant proteins fused to multiple epitopes
AU776576B2 (en) 1999-12-06 2004-09-16 Sangamo Biosciences, Inc. Methods of using randomized libraries of zinc finger proteins for the identification of gene function
US6689558B2 (en) 2000-02-08 2004-02-10 Sangamo Biosciences, Inc. Cells for drug discovery
US20020061512A1 (en) 2000-02-18 2002-05-23 Kim Jin-Soo Zinc finger domains and methods of identifying same
AU2001263155A1 (en) 2000-05-16 2001-11-26 Massachusetts Institute Of Technology Methods and compositions for interaction trap assays
JP2002060786A (ja) 2000-08-23 2002-02-26 Kao Corp 硬質表面用殺菌防汚剤
US7419829B2 (en) 2000-10-06 2008-09-02 Oxford Biomedica (Uk) Limited Vector system
AU2002217929A1 (en) 2000-11-28 2002-06-11 Sangamo Biosciences, Inc. Modulation of gene expression using insulator binding proteins
US7067317B2 (en) 2000-12-07 2006-06-27 Sangamo Biosciences, Inc. Regulation of angiogenesis with zinc finger proteins
WO2002057293A2 (en) 2001-01-22 2002-07-25 Sangamo Biosciences, Inc. Modified zinc finger binding proteins
GB0108491D0 (en) 2001-04-04 2001-05-23 Gendaq Ltd Engineering zinc fingers
JP2005500061A (ja) 2001-08-20 2005-01-06 ザ スクリップス リサーチ インスティテュート Cnnについての亜鉛フィンガー結合ドメイン
US7262054B2 (en) 2002-01-22 2007-08-28 Sangamo Biosciences, Inc. Zinc finger proteins for DNA binding and gene regulation in plants
WO2003087341A2 (en) 2002-01-23 2003-10-23 The University Of Utah Research Foundation Targeted chromosomal mutagenesis using zinc finger nucleases
WO2003080809A2 (en) 2002-03-21 2003-10-02 Sangamo Biosciences, Inc. Methods and compositions for using zinc finger endonucleases to enhance homologous recombination
US7361635B2 (en) 2002-08-29 2008-04-22 Sangamo Biosciences, Inc. Simultaneous modulation of multiple genes
EP2806025B1 (en) 2002-09-05 2019-04-03 California Institute of Technology Use of zinc finger nucleases to stimulate gene targeting
US7888121B2 (en) 2003-08-08 2011-02-15 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
US8409861B2 (en) 2003-08-08 2013-04-02 Sangamo Biosciences, Inc. Targeted deletion of cellular DNA sequences
US20070134796A1 (en) 2005-07-26 2007-06-14 Sangamo Biosciences, Inc. Targeted integration and expression of exogenous nucleic acid sequences
US7972854B2 (en) 2004-02-05 2011-07-05 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
US7253273B2 (en) 2004-04-08 2007-08-07 Sangamo Biosciences, Inc. Treatment of neuropathic pain with zinc finger proteins
AU2005232665B2 (en) 2004-04-08 2010-05-13 Sangamo Therapeutics, Inc. Methods and compositions for treating neuropathic and neurodegenerative conditions
AU2005287278B2 (en) 2004-09-16 2011-08-04 Sangamo Biosciences, Inc. Compositions and methods for protein production
ES2465996T3 (es) 2006-05-25 2014-06-09 Sangamo Biosciences, Inc. Métodos y composiciones para la inactivación genética
EP2027262B1 (en) 2006-05-25 2010-03-31 Sangamo Biosciences Inc. Variant foki cleavage half-domains
DE602008003684D1 (de) 2007-04-26 2011-01-05 Sangamo Biosciences Inc Gezielte integration in die ppp1r12c-position
US8563314B2 (en) 2007-09-27 2013-10-22 Sangamo Biosciences, Inc. Methods and compositions for modulating PD1
CA2720903C (en) 2008-04-14 2019-01-15 Sangamo Biosciences, Inc. Linear donor constructs for targeted integration
WO2009151591A2 (en) 2008-06-10 2009-12-17 Sangamo Biosciences, Inc. Methods and compositions for generation of bax- and bak-deficient cell lines
SG191561A1 (en) 2008-08-22 2013-07-31 Sangamo Biosciences Inc Methods and compositions for targeted single-stranded cleavage and targeted integration
EP2352369B1 (en) 2008-12-04 2017-04-26 Sangamo BioSciences, Inc. Genome editing in rats using zinc-finger nucleases
EP2206723A1 (en) 2009-01-12 2010-07-14 Bonas, Ulla Modular DNA-binding domains
US8586526B2 (en) 2010-05-17 2013-11-19 Sangamo Biosciences, Inc. DNA-binding proteins and uses thereof
US8956828B2 (en) 2009-11-10 2015-02-17 Sangamo Biosciences, Inc. Targeted disruption of T cell receptor genes using engineered zinc finger protein nucleases
ES2751916T3 (es) * 2010-02-08 2020-04-02 Sangamo Therapeutics Inc Semidominios de escisión genomanipulados
EP2660318A1 (en) 2010-02-09 2013-11-06 Sangamo BioSciences, Inc. Targeted genomic modification with partially single-stranded donor molecules
US9567573B2 (en) 2010-04-26 2017-02-14 Sangamo Biosciences, Inc. Genome editing of a Rosa locus using nucleases
HRP20200254T1 (hr) 2010-05-03 2020-05-29 Sangamo Therapeutics, Inc. Pripravci za povezivanje modula cink fingera
AU2011281062B2 (en) 2010-07-21 2015-01-22 Board Of Regents, The University Of Texas System Methods and compositions for modification of a HLA locus
CN102358902B (zh) 2011-04-02 2013-01-02 西南大学 家蚕丝素重链基因突变序列及突变的方法和应用
CA2848417C (en) 2011-09-21 2023-05-02 Sangamo Biosciences, Inc. Methods and compositions for regulation of transgene expression
CA3099582A1 (en) 2011-10-27 2013-05-02 Sangamo Biosciences, Inc. Methods and compositions for modification of the hprt locus
HK1200871A1 (en) 2011-11-16 2015-08-14 Sangamo Therapeutics, Inc. Modified dna-binding proteins and uses thereof
EP3196301B1 (en) 2012-07-11 2018-10-17 Sangamo Therapeutics, Inc. Methods and compositions for the treatment of monogenic diseases
SG10201701601WA (en) * 2012-08-29 2017-04-27 Sangamo Biosciences Inc Methods and compositions for treatment of a genetic condition
US9255250B2 (en) 2012-12-05 2016-02-09 Sangamo Bioscience, Inc. Isolated mouse or human cell having an exogenous transgene in an endogenous albumin gene
US10030063B2 (en) 2012-12-18 2018-07-24 Novartis Ag Production of therapeutic proteins in genetically modified mammalian cells
CN105683376A (zh) 2013-05-15 2016-06-15 桑格摩生物科学股份有限公司 用于治疗遗传病状的方法和组合物
CA3131284C (en) 2013-08-28 2023-09-19 David Paschon Compositions for linking dna-binding domains and cleavage domains
CN105940013B (zh) 2013-12-09 2020-03-27 桑格摩生物科学股份有限公司 用于治疗血友病的方法和组合物
US10179918B2 (en) 2015-05-07 2019-01-15 Sangamo Therapeutics, Inc. Methods and compositions for increasing transgene activity
MY189674A (en) 2015-10-28 2022-02-24 Sangamo Therapeutics Inc Liver-specific constructs, factor viii expression cassettes and methods of use thereof
BR112018012235A2 (pt) 2015-12-18 2018-12-04 Sangamo Therapeutics Inc rompimento alvejado do receptor de células de mhc
WO2017123757A1 (en) * 2016-01-15 2017-07-20 Sangamo Therapeutics, Inc. Methods and compositions for the treatment of neurologic disease
EP3995574A1 (en) 2016-08-24 2022-05-11 Sangamo Therapeutics, Inc. Regulation of gene expression using engineered nucleases
KR102455249B1 (ko) 2016-08-24 2022-10-17 상가모 테라퓨틱스, 인코포레이티드 가공된 표적 특이적 뉴클레아제
AU2017374042C1 (en) 2016-12-09 2024-07-11 Acuitas Therapeutics, Inc. Delivery of target specific nucleases
US20200063160A1 (en) * 2018-08-07 2020-02-27 Sangamo Therapeutics, Inc. Method for the treatment of mucopolysaccharidosis type ii

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