JPWO2019157324A5 - - Google Patents

Download PDF

Info

Publication number
JPWO2019157324A5
JPWO2019157324A5 JP2020542727A JP2020542727A JPWO2019157324A5 JP WO2019157324 A5 JPWO2019157324 A5 JP WO2019157324A5 JP 2020542727 A JP2020542727 A JP 2020542727A JP 2020542727 A JP2020542727 A JP 2020542727A JP WO2019157324 A5 JPWO2019157324 A5 JP WO2019157324A5
Authority
JP
Japan
Prior art keywords
composition
subject
zfn
sequence
aav vector
Prior art date
Legal status (The legal status 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 status listed.)
Granted
Application number
JP2020542727A
Other languages
English (en)
Japanese (ja)
Other versions
JP2021513338A5 (https=
JP7275152B2 (ja
JP2021513338A (ja
Publication date
Application filed filed Critical
Priority claimed from PCT/US2019/017273 external-priority patent/WO2019157324A1/en
Publication of JP2021513338A publication Critical patent/JP2021513338A/ja
Publication of JP2021513338A5 publication Critical patent/JP2021513338A5/ja
Publication of JPWO2019157324A5 publication Critical patent/JPWO2019157324A5/ja
Priority to JP2023076356A priority Critical patent/JP2023090871A/ja
Application granted granted Critical
Publication of JP7275152B2 publication Critical patent/JP7275152B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

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)

Priority Applications (1)

Application Number Priority Date Filing Date Title
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

Related Child Applications (1)

Application Number Title Priority Date Filing Date
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

Publications (4)

Publication Number Publication Date
JP2021513338A JP2021513338A (ja) 2021-05-27
JP2021513338A5 JP2021513338A5 (https=) 2022-02-07
JPWO2019157324A5 true JPWO2019157324A5 (https=) 2022-02-07
JP7275152B2 JP7275152B2 (ja) 2023-05-17

Family

ID=67476462

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2020542727A Active JP7275152B2 (ja) 2018-02-08 2019-02-08 操作された標的特異的なヌクレアーゼ
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

Family Applications After (1)

Application Number Title Priority Date Filing Date
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

Country Status (13)

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

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.
CA3039673A1 (en) 2016-10-20 2018-04-26 Sangamo Therapeutics, Inc. Methods and compositions for the treatment of fabry disease
US11512287B2 (en) * 2017-06-16 2022-11-29 Sangamo Therapeutics, Inc. Targeted disruption of T cell and/or HLA receptors
BR112019026622A2 (pt) * 2017-06-16 2020-06-30 Sangamo Therapeutics, Inc. disrupção direcionada de receptores de célula t e/ou hla
US20200063160A1 (en) * 2018-08-07 2020-02-27 Sangamo Therapeutics, Inc. Method for the treatment of mucopolysaccharidosis type ii
US12290574B2 (en) 2018-08-24 2025-05-06 Spark Therapeutics, Inc. Optimized promoter sequences, intron-free expression constructs and methods of use
JP7716338B2 (ja) * 2019-01-04 2025-07-31 サンガモ セラピューティクス， インコーポレイテッド ファブリー病の処置のための方法及び組成物
EP3826664A4 (en) * 2019-02-06 2022-10-26 Sangamo Therapeutics, Inc. Method for the treatment of mucopolysaccharidosis type i
JP2022553828A (ja) 2019-11-01 2022-12-26 サンガモ セラピューティクス， インコーポレイテッド ゲノム操作のための組成物および方法
WO2021252952A1 (en) * 2020-06-11 2021-12-16 Sangamo Therapeutics, Inc. Methods and compositions for expressing phenylalanine hydroxylase
BR112023018948A2 (pt) * 2021-03-19 2023-10-17 Metagenomi Inc Edição multiplex com enzimas cas
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
WO2023039598A1 (en) * 2021-09-13 2023-03-16 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
US5356802A (en) 1992-04-03 1994-10-18 The Johns Hopkins University Functional domains in flavobacterium okeanokoites (FokI) restriction endonuclease
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
DE69534629D1 (de) 1994-01-18 2005-12-29 Scripps Research Inst Derivate von zinkfingerproteinen und methoden
US6140466A (en) 1994-01-18 2000-10-31 The Scripps Research Institute Zinc finger protein derivatives and methods therefor
US6242568B1 (en) 1994-01-18 2001-06-05 The Scripps Research Institute Zinc finger protein derivatives and methods therefor
GB9824544D0 (en) 1998-11-09 1999-01-06 Medical Res Council Screening system
ATE524545T1 (de) 1994-08-20 2011-09-15 Gendaq Ltd Verbesserungen bei oder im zusammenhang mit bindeproteinen zur erkennung von dna
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
DE69942334D1 (de) 1998-03-02 2010-06-17 Massachusetts Inst Technology Poly-zinkfinger-proteine mit verbesserten linkern
US6140081A (en) 1998-10-16 2000-10-31 The Scripps Research Institute Zinc finger binding domains for GNN
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
US7013219B2 (en) 1999-01-12 2006-03-14 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
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
US6534261B1 (en) 1999-01-12 2003-03-18 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
US6794136B1 (en) 2000-11-20 2004-09-21 Sangamo Biosciences, Inc. Iterative optimization in the design of binding proteins
US7030215B2 (en) 1999-03-24 2006-04-18 Sangamo Biosciences, Inc. Position dependent recognition of GNN nucleotide triplets by zinc fingers
US6379903B1 (en) 1999-10-08 2002-04-30 Sigma-Aldrich Co. Purification of recombinant proteins fused to multiple epitopes
CA2394850C (en) 1999-12-06 2012-02-07 Sangamo Biosciences, Inc. Methods of using randomized libraries of zinc finger proteins for the identification of gene function
KR20020086508A (ko) 2000-02-08 2002-11-18 상가모 바이오사이언스 인코포레이티드 약물 발견용 세포
US20020061512A1 (en) 2000-02-18 2002-05-23 Kim Jin-Soo Zinc finger domains and methods of identifying same
US20030044787A1 (en) 2000-05-16 2003-03-06 Joung J. Keith 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
US20050267061A1 (en) 2004-04-08 2005-12-01 Sangamo Biosciences, Inc. Methods and compositions for treating neuropathic and neurodegenerative conditions
US7067317B2 (en) 2000-12-07 2006-06-27 Sangamo Biosciences, Inc. Regulation of angiogenesis with zinc finger proteins
WO2002057294A2 (en) 2001-01-22 2002-07-25 Sangamo Biosciences, Inc. Zinc finger proteins for dna binding and gene regulation in plants
GB0108491D0 (en) 2001-04-04 2001-05-23 Gendaq Ltd Engineering zinc fingers
US20040224385A1 (en) 2001-08-20 2004-11-11 Barbas Carlos F Zinc finger binding domains for cnn
US7262054B2 (en) 2002-01-22 2007-08-28 Sangamo Biosciences, Inc. Zinc finger proteins for DNA binding and gene regulation in plants
EP1476547B1 (en) 2002-01-23 2006-12-06 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
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
US8409861B2 (en) 2003-08-08 2013-04-02 Sangamo Biosciences, Inc. Targeted deletion of cellular DNA sequences
US7888121B2 (en) 2003-08-08 2011-02-15 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
US7972854B2 (en) 2004-02-05 2011-07-05 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
JP2008506359A (ja) 2004-04-08 2008-03-06 サンガモ バイオサイエンシズ インコーポレイテッド ジンクフィンガータンパク質による神経因性疼痛の処置
KR20070060115A (ko) 2004-09-16 2007-06-12 상가모 바이오사이언스 인코포레이티드 단백질 생산을 위한 조성물 및 방법
AU2006272634B2 (en) 2005-07-26 2013-01-24 Sangamo Therapeutics, Inc. Targeted integration and expression of exogenous nucleic acid sequences
JP5266210B2 (ja) 2006-05-25 2013-08-21 サンガモ バイオサイエンシズ インコーポレイテッド 改変開裂ハーフドメイン
ES2465996T3 (es) 2006-05-25 2014-06-09 Sangamo Biosciences, Inc. Métodos y composiciones para la inactivación genética
WO2008133938A2 (en) 2007-04-26 2008-11-06 Sangamo Biosciences, Inc. Targeted integration into the ppp1r12c locus
US8563314B2 (en) 2007-09-27 2013-10-22 Sangamo Biosciences, Inc. Methods and compositions for modulating PD1
EP2281050B1 (en) 2008-04-14 2014-04-02 Sangamo BioSciences, Inc. Linear donor constructs for targeted integration
KR101802393B1 (ko) 2008-06-10 2017-11-28 상가모 테라퓨틱스, 인코포레이티드 Ｂａｘ- 및 ｂａｋ-결함 세포주들의 제조 방법 및 조성물
SG191561A1 (en) 2008-08-22 2013-07-31 Sangamo Biosciences Inc Methods and compositions for targeted single-stranded cleavage and targeted integration
EP3156494B8 (en) 2008-12-04 2018-09-19 Sangamo Therapeutics, Inc. Genome editing in rats using zinc-finger nucleases
EP2206723A1 (en) 2009-01-12 2010-07-14 Bonas, Ulla Modular DNA-binding domains
US8956828B2 (en) 2009-11-10 2015-02-17 Sangamo Biosciences, Inc. Targeted disruption of T cell receptor genes using engineered zinc finger protein nucleases
EP3594333B1 (en) 2010-02-08 2023-11-01 Sangamo Therapeutics, Inc. Engineered cleavage half-domains
EP2534163B1 (en) 2010-02-09 2015-11-04 Sangamo BioSciences, Inc. Targeted genomic modification with partially single-stranded donor molecules
US8771985B2 (en) 2010-04-26 2014-07-08 Sangamo Biosciences, Inc. Genome editing of a Rosa locus using zinc-finger nucleases
PT2566972T (pt) 2010-05-03 2020-03-02 Sangamo Biosciences Inc Composições para a ligação de módulos de dedo de zinco
JP6208580B2 (ja) 2010-05-17 2017-10-04 サンガモ セラピューティクス， インコーポレイテッド 新規のｄｎａ結合タンパク質及びその使用
CA2993567C (en) 2010-07-21 2022-06-28 Sangamo Biosciences, Inc. Methods and compositions for modification of a t-cell receptor gene
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
AU2012312260B2 (en) 2011-09-21 2017-08-31 Sangamo Therapeutics, Inc. Methods and compositions for regulation of transgene expression
JP6188703B2 (ja) 2011-10-27 2017-08-30 サンガモ セラピューティクス， インコーポレイテッド Ｈｐｒｔ遺伝子座を修飾するための方法および組成物
AU2012340213B2 (en) 2011-11-16 2017-12-07 Sangamo Therapeutics, Inc. Modified DNA-binding proteins and uses thereof
DK3444342T3 (da) 2012-07-11 2020-08-24 Sangamo Therapeutics Inc Fremgangsmåder og sammensætninger til behandlingen af lysosomale aflejringssygdomme
SI2890780T1 (sl) * 2012-08-29 2020-11-30 Sangamo Therapeutics, Inc. Postopki in sestavki za zdravljenje genetskega stanja
WO2014089212A1 (en) 2012-12-05 2014-06-12 Sangamo Biosciences, Inc. Methods and compositions for regulation of metabolic disorders
WO2014097113A2 (en) * 2012-12-18 2014-06-26 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
AU2014312295C1 (en) 2013-08-28 2020-08-13 Sangamo Therapeutics, Inc. Compositions for linking DNA-binding domains and cleavage domains
DK3080274T3 (da) 2013-12-09 2020-08-31 Sangamo Therapeutics Inc Fremgangsmåder og sammensætninger til genom-manipulation
US10179918B2 (en) 2015-05-07 2019-01-15 Sangamo Therapeutics, Inc. Methods and compositions for increasing transgene activity
CA3002524A1 (en) 2015-10-28 2017-05-04 Sangamo Therapeutics, Inc. Liver-specific constructs, factor viii expression cassettes and methods of use thereof
IL297018A (en) 2015-12-18 2022-12-01 Sangamo Therapeutics Inc Directed cleavage of cell mhc receptor
CA3010738A1 (en) * 2016-01-15 2017-07-20 Sangamo Therapeutics, Inc. Methods and compositions for the treatment of neurologic disease
LT3504229T (lt) 2016-08-24 2021-12-10 Sangamo Therapeutics, Inc. Genų raiškos reguliavimas, panaudojant rekombinantines nukleazes
SG10201913948PA (en) 2016-08-24 2020-03-30 Sangamo Therapeutics Inc Engineered target specific nucleases
EP3551169A4 (en) 2016-12-09 2020-09-09 Sangamo Therapeutics, Inc. TARGET-SPECIFIC NUCLEASES ADMINISTRATION
US20200063160A1 (en) * 2018-08-07 2020-02-27 Sangamo Therapeutics, Inc. Method for the treatment of mucopolysaccharidosis type ii

Similar Documents

Publication Publication Date Title
JP2021513338A5 (https=)
RU2020129448A (ru) Сконструированные специфические для мишени нуклеазы
JP7837302B2 (ja) 治療用途のための微小胞および幹細胞組成物
JPWO2019157324A5 (https=)
US20230118337A1 (en) Compositions and methods for the treatment of hemoglobinopathies
US11479775B2 (en) RNA targeting of mutations via suppressor tRNAs and deaminases
Almeida-Porada et al. Therapeutic mesenchymal stromal cells for immunotherapy and for gene and drug delivery
Parascandolo et al. Carcinogenesis and reactive oxygen species signaling: interaction of the NADPH oxidase NOX1–5 and superoxide dismutase 1–3 signal transduction pathways
JP2017504354A5 (https=)
JP2020500162A5 (https=)
JP2017500061A5 (https=)
Beagan et al. Drosophila DNA polymerase theta utilizes both helicase-like and polymerase domains during microhomology-mediated end joining and interstrand crosslink repair
JP2018531614A5 (https=)
JP2015533786A5 (https=)
Benoit et al. A novel highly divergent protein family identified from a viviparous insect by RNA-seq analysis: a potential target for tsetse fly-specific abortifacients
JP2016539627A5 (https=)
CN104284669A (zh) 治疗血红蛋白病的组合物和方法
Locatelli et al. Autologous gene therapy for hemoglobinopathies: From bench to patient’s bedside
Bueren et al. Advances in the gene therapy of monogenic blood cell diseases
GB2560250A (en) ATP-Binding cassette family coding polyribonucleotides and formulations thereof
Kyriakopoulou et al. Gene editing innovations and their applications in cardiomyopathy research
CN119923466A (zh) 用于减少补体激活的组合物和方法
JPWO2020160514A5 (https=)
Kato et al. Molecular cloning and expression of mouse Mg2+-dependent protein phosphatase β-4 (type 2Cβ-4)
Tatsumi et al. 2′, 5′-oligoadenylate synthetase gene in chicken: gene structure, distribution of alleles and their expression