JP5551432B2 - 遺伝子不活性化のための方法と組成物 - Google Patents

遺伝子不活性化のための方法と組成物 Download PDF

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JP5551432B2
JP5551432B2 JP2009512175A JP2009512175A JP5551432B2 JP 5551432 B2 JP5551432 B2 JP 5551432B2 JP 2009512175 A JP2009512175 A JP 2009512175A JP 2009512175 A JP2009512175 A JP 2009512175A JP 5551432 B2 JP5551432 B2 JP 5551432B2
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JP2009538141A5 (enExample
JP2009538141A (ja
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アンドー,デール
クリストファー ホームズ,マイケル
カ レオン リー,ゲアリー
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Sangamo Therapeutics Inc
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JP2009512175A 2006-05-25 2007-05-23 遺伝子不活性化のための方法と組成物 Expired - Fee Related JP5551432B2 (ja)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US80850106P 2006-05-25 2006-05-25
US60/808,501 2006-05-25
US84726906P 2006-09-26 2006-09-26
US60/847,269 2006-09-26
US92691107P 2007-04-30 2007-04-30
US60/926,911 2007-04-30
PCT/US2007/012588 WO2007139982A2 (en) 2006-05-25 2007-05-23 Methods and compositions for gene inactivation

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JP2009538141A5 JP2009538141A5 (enExample) 2010-03-04
JP5551432B2 true JP5551432B2 (ja) 2014-07-16

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US (7) US7951925B2 (enExample)
EP (4) EP2019839B1 (enExample)
JP (2) JP5551432B2 (enExample)
AT (1) ATE536371T1 (enExample)
AU (1) AU2007267874B2 (enExample)
CA (1) CA2651499C (enExample)
ES (2) ES2378333T3 (enExample)
WO (1) WO2007139982A2 (enExample)

Families Citing this family (229)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US8278079B2 (en) * 2002-11-07 2012-10-02 Danisco Us Inc. BGL6 β-glucosidase and nucleic acids encoding the same
US20120196370A1 (en) 2010-12-03 2012-08-02 Fyodor Urnov Methods and compositions for targeted genomic deletion
JP5551432B2 (ja) 2006-05-25 2014-07-16 サンガモ バイオサイエンシーズ, インコーポレイテッド 遺伝子不活性化のための方法と組成物
EP2089427B1 (en) 2006-11-13 2014-07-30 Sangamo BioSciences, Inc. Zinc finger nuclease for targeting the human glucocorticoid receptor locus
AU2008262487B2 (en) * 2007-05-23 2013-10-31 Sangamo Therapeutics, Inc. Methods and compositions for increased transgene expression
US11235026B2 (en) 2007-09-27 2022-02-01 Sangamo Therapeutics, Inc. Methods and compositions for modulating PD1
EP2188384B1 (en) * 2007-09-27 2015-07-15 Sangamo BioSciences, Inc. Rapid in vivo identification of biologically active nucleases
US8563314B2 (en) 2007-09-27 2013-10-22 Sangamo Biosciences, Inc. Methods and compositions for modulating PD1
EP2205752B1 (en) * 2007-10-25 2016-08-10 Sangamo BioSciences, Inc. Methods and compositions for targeted integration
US8791324B2 (en) 2008-04-21 2014-07-29 Danziger Innovations Ltd. Plant viral expression vectors and use of same for generating genotypic variations in plant genomes
WO2009154686A1 (en) 2008-05-28 2009-12-23 Sangamo Biosciences, Inc. Compositions for linking dna-binding domains and cleavage domains
KR101802393B1 (ko) * 2008-06-10 2017-11-28 상가모 테라퓨틱스, 인코포레이티드 Bax- 및 bak-결함 세포주들의 제조 방법 및 조성물
US8703489B2 (en) 2008-08-22 2014-04-22 Sangamo Biosciences, Inc. Methods and compositions for targeted single-stranded cleavage and targeted integration
KR101673566B1 (ko) * 2008-10-29 2016-11-07 상가모 바이오사이언스 인코포레이티드 글루타민 신테타제 유전자 발현을 불활성화시키기 위한 방법 및 조성물
US20110023153A1 (en) * 2008-12-04 2011-01-27 Sigma-Aldrich Co. Genomic editing of genes involved in alzheimer's disease
US20110023139A1 (en) * 2008-12-04 2011-01-27 Sigma-Aldrich Co. Genomic editing of genes involved in cardiovascular disease
US20110030072A1 (en) * 2008-12-04 2011-02-03 Sigma-Aldrich Co. Genome editing of immunodeficiency genes in animals
US20110023146A1 (en) * 2008-12-04 2011-01-27 Sigma-Aldrich Co. Genomic editing of genes involved in secretase-associated disorders
US20110016541A1 (en) * 2008-12-04 2011-01-20 Sigma-Aldrich Co. Genome editing of sensory-related genes in animals
US20110023149A1 (en) * 2008-12-04 2011-01-27 Sigma-Aldrich Co. Genomic editing of genes involved in tumor suppression in animals
US20110016543A1 (en) * 2008-12-04 2011-01-20 Sigma-Aldrich Co. Genomic editing of genes involved in inflammation
US20110023140A1 (en) * 2008-12-04 2011-01-27 Sigma-Aldrich Co. Rabbit genome editing with zinc finger nucleases
US20110023158A1 (en) * 2008-12-04 2011-01-27 Sigma-Aldrich Co. Bovine genome editing with zinc finger nucleases
US20110023151A1 (en) * 2008-12-04 2011-01-27 Sigma-Aldrich Co. Genome editing of abc transporters
US20110023148A1 (en) * 2008-12-04 2011-01-27 Sigma-Aldrich Co. Genome editing of addiction-related genes in animals
US20110023141A1 (en) * 2008-12-04 2011-01-27 Sigma-Aldrich Co. Genomic editing of genes involved with parkinson's disease
US20110016540A1 (en) * 2008-12-04 2011-01-20 Sigma-Aldrich Co. Genome editing of genes associated with trinucleotide repeat expansion disorders in animals
ES2627552T3 (es) 2008-12-04 2017-07-28 Sigma Aldrich Company Edición de genoma en ratas usando nucleasas con dedos de cinc
US20110023145A1 (en) * 2008-12-04 2011-01-27 Sigma-Aldrich Co. Genomic editing of genes involved in autism spectrum disorders
US20110023150A1 (en) * 2008-12-04 2011-01-27 Sigma-Aldrich Co. Genome editing of genes associated with schizophrenia in animals
US20110023156A1 (en) * 2008-12-04 2011-01-27 Sigma-Aldrich Co. Feline genome editing with zinc finger nucleases
US20110016546A1 (en) * 2008-12-04 2011-01-20 Sigma-Aldrich Co. Porcine genome editing with zinc finger nucleases
US20110023152A1 (en) * 2008-12-04 2011-01-27 Sigma-Aldrich Co. Genome editing of cognition related genes in animals
US20110023144A1 (en) * 2008-12-04 2011-01-27 Sigma-Aldrich Co. Genomic editing of genes involved in amyotrophyic lateral sclerosis disease
US20110016539A1 (en) * 2008-12-04 2011-01-20 Sigma-Aldrich Co. Genome editing of neurotransmission-related genes in animals
US20110023143A1 (en) * 2008-12-04 2011-01-27 Sigma-Aldrich Co. Genomic editing of neurodevelopmental genes in animals
US20110023147A1 (en) * 2008-12-04 2011-01-27 Sigma-Aldrich Co. Genomic editing of prion disorder-related genes in animals
US20110023154A1 (en) * 2008-12-04 2011-01-27 Sigma-Aldrich Co. Silkworm genome editing with zinc finger nucleases
US8871905B2 (en) * 2009-03-20 2014-10-28 Sangamo Biosciences, Inc. Modification of CXCR4 using engineered zinc finger proteins
CA2756833C (en) 2009-04-09 2019-11-19 Sangamo Biosciences, Inc. Targeted integration into stem cells
US8772008B2 (en) 2009-05-18 2014-07-08 Sangamo Biosciences, Inc. Methods and compositions for increasing nuclease activity
CA2765488C (en) * 2009-06-30 2018-01-02 Sangamo Biosciences, Inc. Rapid screening of biologically active nucleases and isolation of nuclease-modified cells
US9234016B2 (en) * 2009-07-28 2016-01-12 Sangamo Biosciences, Inc. Engineered zinc finger proteins for treating trinucleotide repeat disorders
US9476060B2 (en) 2009-10-21 2016-10-25 Danziger Innovations Ltd. Generating genotypic variations in plant genomes by gamete infection
US8956828B2 (en) * 2009-11-10 2015-02-17 Sangamo Biosciences, Inc. Targeted disruption of T cell receptor genes using engineered zinc finger protein nucleases
WO2011064750A1 (en) 2009-11-27 2011-06-03 Basf Plant Science Company Gmbh Chimeric endonucleases and uses thereof
US9493827B2 (en) * 2010-01-14 2016-11-15 Deutsches Krebsforschungszentrum Determination of in vivo DNA double-strand break localization and application thereof
PH12012501491B1 (en) * 2010-01-22 2019-06-28 Dow Agrosciences Llc Targeted genomic alteration
EP2534173B1 (en) 2010-02-08 2019-09-11 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
CN102939377B (zh) 2010-04-26 2016-06-08 桑格摩生物科学股份有限公司 使用锌指核酸酶对Rosa位点进行基因组编辑
WO2011139349A1 (en) 2010-05-03 2011-11-10 Sangamo Biosciences, Inc. Compositions for linking zinc finger modules
EP2571512B1 (en) 2010-05-17 2017-08-23 Sangamo BioSciences, Inc. Novel dna-binding proteins and uses thereof
US20110201118A1 (en) * 2010-06-14 2011-08-18 Iowa State University Research Foundation, Inc. Nuclease activity of tal effector and foki fusion protein
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
WO2012012738A1 (en) * 2010-07-23 2012-01-26 Sigma-Aldrich Co., Llc Genome editing using targeting endonucleases and single-stranded nucleic acids
WO2012015938A2 (en) * 2010-07-27 2012-02-02 The Johns Hopkins University Obligate heterodimer variants of foki cleavage domain
WO2012018726A1 (en) * 2010-08-02 2012-02-09 Cellectis Sa Method for increasing double-strand break-induced gene targeting
EP3511420B1 (en) 2010-09-27 2021-04-07 Sangamo Therapeutics, Inc. Compositions for inhibiting viral entry into cells
US9175280B2 (en) 2010-10-12 2015-11-03 Sangamo Biosciences, Inc. Methods and compositions for treating hemophilia B
ES2713560T3 (es) 2010-10-15 2019-05-22 Fund Centre De Regulacio Genòmica Péptidos que tienen dominios de dedos de zinc y usos de estos
CN102234628A (zh) * 2011-05-06 2011-11-09 中国科学院广州生物医药与健康研究院 Ccr5缺失型的造血干细胞及其制备方法与应用
EP2522726A1 (en) 2011-05-12 2012-11-14 Fundació Privada Centre de Regulació Genòmica (CRG) Zinc finger nucleases for p53 editing
KR102061557B1 (ko) 2011-09-21 2020-01-03 상가모 테라퓨틱스, 인코포레이티드 이식 유전자 발현의 조절을 위한 방법 및 조성물
EP2758537A4 (en) * 2011-09-23 2015-08-12 Univ Iowa State Res Found MONOERARY TECHNIQUE OF TAL NUCLEASE OR ZINC FINGERUCLEASE FOR DNA MODIFICATION
AU2012328682B2 (en) * 2011-10-27 2017-09-21 Sangamo Therapeutics, Inc. Methods and compositions for modification of the HPRT locus
CA2854819C (en) 2011-11-16 2022-07-19 Sangamo Biosciences, Inc. Modified dna-binding proteins and uses thereof
US9688997B2 (en) 2011-12-29 2017-06-27 Iowa State University Research Foundation, Inc. Genetically modified plants with resistance to Xanthomonas and other bacterial plant pathogens
GB201122458D0 (en) 2011-12-30 2012-02-08 Univ Wageningen Modified cascade ribonucleoproteins and uses thereof
HK1203566A1 (zh) 2012-02-29 2015-10-30 桑格摩生物科学股份有限公司 治療亨廷頓氏病的方法和組合物
EP2847338B1 (en) 2012-05-07 2018-09-19 Sangamo Therapeutics, Inc. Methods and compositions for nuclease-mediated targeted integration of transgenes
MY189533A (en) 2012-05-25 2022-02-16 Univ California Methods and compositions for rna-directed target dna modification and for rna-directed modulation of transcription
US10648001B2 (en) 2012-07-11 2020-05-12 Sangamo Therapeutics, Inc. Method of treating mucopolysaccharidosis type I or II
EP3816281A1 (en) 2012-07-11 2021-05-05 Sangamo Therapeutics, Inc. Methods and compositions for the treatment of lysosomal storage diseases
US20150104873A1 (en) * 2012-07-11 2015-04-16 University of Nevada, Las Vegas Genome Surgery with Paired, Permeant Endonuclease Construct
JP6329537B2 (ja) 2012-07-11 2018-05-23 サンガモ セラピューティクス, インコーポレイテッド 生物学的薬剤の送達のための方法および組成物
KR20220164090A (ko) 2012-08-29 2022-12-12 상가모 테라퓨틱스, 인코포레이티드 유전적 병태를 치료하기 위한 방법 및 조성물
ES2824024T3 (es) 2012-10-10 2021-05-11 Sangamo Therapeutics Inc Compuestos modificadores de células T y usos de los mismos
US20140140969A1 (en) * 2012-11-20 2014-05-22 Sangamo Biosciences, Inc. Methods and compositions for muscular dystrophies
AU2013355327A1 (en) 2012-12-05 2015-06-11 Sangamo Therapeutics, Inc. Methods and compositions for regulation of metabolic disorders
AU2013355214B2 (en) 2012-12-06 2017-06-15 Sigma-Aldrich Co. Llc Crispr-based genome modification and regulation
US10227610B2 (en) 2013-02-25 2019-03-12 Sangamo Therapeutics, Inc. Methods and compositions for enhancing nuclease-mediated gene disruption
WO2014150624A1 (en) 2013-03-14 2014-09-25 Caribou Biosciences, Inc. Compositions and methods of nucleic acid-targeting nucleic acids
JP6346266B2 (ja) 2013-03-21 2018-06-20 サンガモ セラピューティクス, インコーポレイテッド 操作されたジンクフィンガータンパク質ヌクレアーゼを使用するt細胞受容体遺伝子の標的化された破壊
WO2014165825A2 (en) * 2013-04-04 2014-10-09 President And Fellows Of Harvard College Therapeutic uses of genome editing with crispr/cas systems
AU2014262867B2 (en) * 2013-05-10 2019-12-05 Sangamo Therapeutics, Inc. Delivery methods and compositions for nuclease-mediated genome engineering
AU2014265331B2 (en) 2013-05-15 2019-12-05 Sangamo Therapeutics, Inc. Methods and compositions for treatment of a genetic condition
EP3591045B1 (en) 2013-08-28 2024-07-17 Sangamo Therapeutics, Inc. Compositions for linking dna-binding domains and cleavage domains
DK3441468T3 (da) 2013-10-17 2021-07-26 Sangamo Therapeutics Inc Afgivelsesfremgangsmåder og sammensætninger til nukleasemedieret genom-manipulation
WO2015057976A1 (en) 2013-10-17 2015-04-23 Sangamo Biosciences, Inc. Delivery methods and compositions for nuclease-mediated genome engineering in hematopoietic stem cells
JP2016537341A (ja) 2013-11-11 2016-12-01 サンガモ バイオサイエンシーズ, インコーポレイテッド ハンチントン病を処置するための方法および組成物
PT3492593T (pt) 2013-11-13 2021-10-18 Childrens Medical Center Regulação da expressão de genes mediada por nucleases
EP3760719A1 (en) * 2013-11-18 2021-01-06 CRISPR Therapeutics AG Crispr-cas system materials and methods
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
HRP20201038T1 (hr) 2014-02-03 2020-10-16 Sangamo Therapeutics, Inc. Postupci i pripravci za liječenje beta talasemije
US10370680B2 (en) 2014-02-24 2019-08-06 Sangamo Therapeutics, Inc. Method of treating factor IX deficiency using nuclease-mediated targeted integration
KR101576728B1 (ko) * 2014-03-06 2015-12-10 가톨릭대학교 산학협력단 면역세포 백신 및 이의 용도
WO2015143046A2 (en) 2014-03-18 2015-09-24 Sangamo Biosciences, Inc. Methods and compositions for regulation of zinc finger protein expression
US9522936B2 (en) 2014-04-24 2016-12-20 Sangamo Biosciences, Inc. Engineered transcription activator like effector (TALE) proteins
JP2017515819A (ja) 2014-05-08 2017-06-15 サンガモ セラピューティクス, インコーポレイテッド ハンチントン病を処置するための方法および組成物
AU2015259191B2 (en) 2014-05-13 2019-03-21 Sangamo Therapeutics, Inc. Methods and compositions for prevention or treatment of a disease
WO2015188065A1 (en) 2014-06-05 2015-12-10 Sangamo Biosciences, Inc. Methods and compositions for nuclease design
US9816074B2 (en) 2014-07-25 2017-11-14 Sangamo Therapeutics, Inc. Methods and compositions for modulating nuclease-mediated genome engineering in hematopoietic stem cells
US9757420B2 (en) 2014-07-25 2017-09-12 Sangamo Therapeutics, Inc. Gene editing for HIV gene therapy
US9616090B2 (en) 2014-07-30 2017-04-11 Sangamo Biosciences, Inc. Gene correction of SCID-related genes in hematopoietic stem and progenitor cells
WO2016037162A1 (en) 2014-09-07 2016-03-10 Selecta Biosciences, Inc. Methods and compositions for attenuating anti-viral transfer vector immune responses
WO2016044416A1 (en) 2014-09-16 2016-03-24 Sangamo Biosciences, Inc. Methods and compositions for nuclease-mediated genome engineering and correction in hematopoietic stem cells
SG11201703148TA (en) 2014-11-05 2017-05-30 Voyager Therapeutics Inc Aadc polynucleotides for the treatment of parkinson's disease
US10597660B2 (en) 2014-11-14 2020-03-24 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (ALS)
MX2017006217A (es) 2014-11-14 2018-05-02 Voyager Therapeutics Inc Polinucleotidos moduladores.
EP3025719B1 (en) 2014-11-26 2018-09-26 Miltenyi Biotec GmbH Combination immunotherapy of antigen-recognizing receptors and hematopoietic cells for the treatment of diseases
JP2017535296A (ja) 2014-11-27 2017-11-30 ダンツィガー イノベイションズ リミテッドDanziger Innovations Ltd. ゲノム編集のための核酸構築物
US10889834B2 (en) 2014-12-15 2021-01-12 Sangamo Therapeutics, Inc. Methods and compositions for enhancing targeted transgene integration
WO2016118726A2 (en) 2015-01-21 2016-07-28 Sangamo Biosciences, Inc. Methods and compositions for identification of highly specific nucleases
ES2777534T3 (es) 2015-02-13 2020-08-05 Inserm - Institut National De La Santé Et De La Rech Médicale Polipéptidos para la ingeniería de proteínas integrasas quiméricas y su uso en terapia génica
JP2018513681A (ja) 2015-03-31 2018-05-31 エクセリゲン サイエンティフィック, インコーポレイテッドExeligen Scientific, Inc. 細胞または生物のゲノムへのDNA配列の標的化組み込みのためのCas9レトロウイルスインテグラーゼおよびCas9レコンビナーゼ系
EP3277823B1 (en) * 2015-04-03 2023-09-13 Dana-Farber Cancer Institute, Inc. Composition and methods of genome editing of b-cells
US10179918B2 (en) 2015-05-07 2019-01-15 Sangamo Therapeutics, Inc. Methods and compositions for increasing transgene activity
JP2018515139A (ja) 2015-05-08 2018-06-14 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 万能ドナー幹細胞および関連する方法
US10808020B2 (en) * 2015-05-12 2020-10-20 Sangamo Therapeutics, Inc. Nuclease-mediated regulation of gene expression
US11535871B2 (en) * 2015-05-14 2022-12-27 University Of Southern California Optimized gene editing utilizing a recombinant endonuclease system
US9957501B2 (en) 2015-06-18 2018-05-01 Sangamo Therapeutics, Inc. Nuclease-mediated regulation of gene expression
AU2016291778B2 (en) 2015-07-13 2021-05-06 Sangamo Therapeutics, Inc. Delivery methods and compositions for nuclease-mediated genome engineering
PT3352776T (pt) 2015-09-23 2025-06-27 Sangamo Therapeutics Inc Repressores de htt e utilizações dos mesmos
WO2017053729A1 (en) 2015-09-25 2017-03-30 The Board Of Trustees Of The Leland Stanford Junior University Nuclease-mediated genome editing of primary cells and enrichment thereof
EP3368664A4 (en) 2015-10-28 2019-08-28 Sangamo Therapeutics, Inc. LIFE-SPECIFIC CONSTRUCTS, FACTOR VIII EXPRESSION CASSETTES AND METHOD OF USE THEREOF
WO2017079428A1 (en) 2015-11-04 2017-05-11 President And Fellows Of Harvard College Site specific germline modification
AU2016361350B2 (en) 2015-11-23 2023-04-06 Sangamo Therapeutics, Inc. Methods and compositions for engineering immunity
WO2017106528A2 (en) 2015-12-18 2017-06-22 Sangamo Biosciences, Inc. Targeted disruption of the t cell receptor
KR20180088911A (ko) 2015-12-18 2018-08-07 상가모 테라퓨틱스, 인코포레이티드 Mhc 세포 수용체의 표적화된 붕괴
ES2895430T3 (es) 2016-01-15 2022-02-21 Univ Minnesota Métodos y composiciones para el tratamiento de enfermedad neurológica
JP7012650B2 (ja) 2016-02-02 2022-01-28 サンガモ セラピューティクス, インコーポレイテッド Dna結合ドメインと切断ドメインとを連結するための組成物
MX2018014152A (es) 2016-05-18 2019-03-28 Voyager Therapeutics Inc Composiciones y métodos para tratar la enfermedad de huntington.
KR20240056729A (ko) 2016-05-18 2024-04-30 보이저 테라퓨틱스, 인크. 조절성 폴리뉴클레오티드
HUE056707T2 (hu) 2016-08-24 2022-03-28 Sangamo Therapeutics Inc Génexpresszió szabályozása szerkezeti nukleázok alkalmazásával
KR20220145913A (ko) 2016-08-24 2022-10-31 상가모 테라퓨틱스, 인코포레이티드 가공된 표적 특이적 뉴클레아제
CA3035534A1 (en) 2016-09-07 2018-03-15 Sangamo Therapeutics, Inc. Modulation of liver genes
EP4667565A2 (en) 2016-10-20 2025-12-24 Sangamo Therapeutics, Inc. Methods and compositions for the treatment of fabry disease
WO2018081775A1 (en) 2016-10-31 2018-05-03 Sangamo Therapeutics, Inc. Gene correction of scid-related genes in hematopoietic stem and progenitor cells
CN116693411A (zh) 2017-04-28 2023-09-05 爱康泰生治疗公司 用于递送核酸的新型羰基脂质和脂质纳米颗粒制剂
CA3059793A1 (en) 2017-05-03 2018-11-08 Sangamo Therapeutics, Inc. Methods and compositions for modification of a cystic fibrosis transmembrane conductance regulator (cftr) gene
AU2018261003A1 (en) 2017-05-05 2019-11-14 Voyager Therapeutics, Inc. Compositions and methods of treating Huntington's Disease
AU2018261790B2 (en) 2017-05-05 2024-10-03 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (ALS)
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
US11512287B2 (en) 2017-06-16 2022-11-29 Sangamo Therapeutics, Inc. Targeted disruption of T cell and/or HLA receptors
EP3585160A2 (en) 2017-07-31 2020-01-01 Regeneron Pharmaceuticals, Inc. Crispr reporter non-human animals and uses thereof
CA3067872A1 (en) 2017-07-31 2019-02-07 Regeneron Pharmaceuticals, Inc. Cas-transgenic mouse embryonic stem cells and mice and uses thereof
CA3065579A1 (en) 2017-07-31 2019-02-07 Regeneron Pharmaceuticals, Inc. Assessment of crispr/cas-induced recombination with an exogenous donor nucleic acid in vivo
MX2020003838A (es) 2017-10-13 2020-08-06 Selecta Biosciences Inc Metodos y composiciones para atenuar las respuestas de inmunoglobulina m del vector de transferencia antiviral.
JP2021502085A (ja) 2017-11-09 2021-01-28 サンガモ セラピューティクス, インコーポレイテッド サイトカイン誘導性sh2含有タンパク質(cish)遺伝子の遺伝子改変
RU2020129448A (ru) 2018-02-08 2022-03-09 Сангамо Терапьютикс, Инк. Сконструированные специфические для мишени нуклеазы
CA3091143A1 (en) 2018-02-15 2019-08-22 Memorial Sloan-Kettering Cancer Center Foxp3 targeting agent compositions and methods of use for adoptive cell therapy
JP2021515037A (ja) 2018-02-26 2021-06-17 アントルクス,インコーポレーテッド 寛容原性リポソーム及びその使用方法
EP3592140A1 (en) 2018-03-19 2020-01-15 Regeneron Pharmaceuticals, Inc. Transcription modulation in animals using crispr/cas systems
KR20210029707A (ko) 2018-04-05 2021-03-16 주노 쎄러퓨티크스 인코퍼레이티드 재조합 수용체를 발현하는 세포의 생산 방법 및 관련 조성물
CA3095084A1 (en) 2018-04-05 2019-10-10 Juno Therapeutics, Inc. T cells expressing a recombinant receptor, related polynucleotides and methods
CN112313246B (zh) 2018-04-18 2025-05-13 桑格摩生物治疗股份有限公司 用于调节亨廷顿蛋白(htt)的锌指蛋白组合物
EP3794126A1 (en) 2018-05-15 2021-03-24 Voyager Therapeutics, Inc. Compositions and methods for the treatment of parkinson's disease
US11690921B2 (en) 2018-05-18 2023-07-04 Sangamo Therapeutics, Inc. Delivery of target specific nucleases
KR102712986B1 (ko) 2018-08-23 2024-10-07 상가모 테라퓨틱스, 인코포레이티드 조작된 표적 특이적 염기 편집기
EP3849565A4 (en) 2018-09-12 2022-12-28 Fred Hutchinson Cancer Research Center REDUCTION OF CD33 EXPRESSION TO SELECTIVELY PROTECT THERAPEUTIC CELLS
JP2022500052A (ja) 2018-09-18 2022-01-04 サンガモ セラピューティクス, インコーポレイテッド プログラム細胞死1(pd1)特異的ヌクレアーゼ
US12329857B2 (en) 2018-09-21 2025-06-17 Acuitas Therapeutics, Inc. Systems and methods for manufacturing lipid nanoparticles and liposomes
WO2020069029A1 (en) 2018-09-26 2020-04-02 Emendobio Inc. Novel crispr nucleases
JP7633155B2 (ja) * 2018-10-11 2025-02-19 オスペダーレ サン ラファエレ エス.アール.エル 人工トランス活性化因子による選択
EP3867376A1 (en) 2018-10-18 2021-08-25 Intellia Therapeutics, Inc. Nucleic acid constructs and methods of use
MX2021004277A (es) 2018-10-18 2021-09-08 Intellia Therapeutics Inc Composiciones y metodos para expresar el factor ix.
KR20210094609A (ko) 2018-11-28 2021-07-29 포티 세븐, 인코포레이티드 절제 레짐에 대해 저항성인 유전적으로 변형된 hspc
AU2020205717B2 (en) 2019-01-11 2025-09-11 Acuitas Therapeutics, Inc. Lipids for lipid nanoparticle delivery of active agents
US20220098621A1 (en) 2019-02-05 2022-03-31 Emendobio Inc. Crispr compositions and methods for promoting gene editing of ribosomal protein s19 (rps19) gene
WO2020163307A1 (en) 2019-02-06 2020-08-13 Emendobio Inc. New engineered high fidelity cas9
SG11202108451VA (en) 2019-04-03 2021-09-29 Regeneron Pharma Methods and compositions for insertion of antibody coding sequences into a safe harbor locus
MA55811A (fr) 2019-05-01 2022-03-09 Editas Medicine Inc Cellules exprimant un récepteur recombinant à base d'un locus modifié du tgfbr2, et polynucléotides et méthodes associés
JP7754722B2 (ja) 2019-05-01 2025-10-15 ジュノー セラピューティクス インコーポレイテッド 改変されたcd247遺伝子座からキメラ受容体を発現する細胞、関連ポリヌクレオチド、および方法
CN114206396A (zh) 2019-05-28 2022-03-18 西莱克塔生物科技公司 用于减弱抗病毒转移载体免疫应答的方法和组合物
CN114729383A (zh) 2019-07-02 2022-07-08 弗莱德哈钦森癌症研究中心 重组ad35载体及相关基因疗法改进
CA3144413A1 (en) * 2019-07-17 2021-01-21 Rafick-Pierre Sekaly Long lived t cells for treating hiv infection
US20230416776A1 (en) 2019-10-08 2023-12-28 Regents Of The University Of Minnesota Crispr-mediated human genome editing with vectors
US20230001019A1 (en) 2019-11-08 2023-01-05 Regeneron Pharmaceuticals, Inc. Crispr and aav strategies for x-linked juvenile retinoschisis therapy
WO2021108363A1 (en) 2019-11-25 2021-06-03 Regeneron Pharmaceuticals, Inc. Crispr/cas-mediated upregulation of humanized ttr allele
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
US20230181641A1 (en) 2020-05-13 2023-06-15 Juno Therapeutics, Inc. Process for producing donor-batched cells expressing a recombinant receptor
GB202007577D0 (en) 2020-05-21 2020-07-08 Oxford Genetics Ltd Hdr enhancers
EP4153741A1 (en) 2020-05-21 2023-03-29 Oxford Genetics Limited Hdr enhancers
GB202007578D0 (en) 2020-05-21 2020-07-08 Univ Oxford Innovation Ltd Hdr enhancers
US20230183750A1 (en) 2020-05-21 2023-06-15 Oxford Genetics Limited Hdr enhancers
EP4171616A1 (en) 2020-06-26 2023-05-03 Juno Therapeutics GmbH Engineered t cells conditionally expressing a recombinant receptor, related polynucleotides and methods
GB202010075D0 (en) 2020-07-01 2020-08-12 Imp College Innovations Ltd Therapeutic nucleic acids, peptides and uses
WO2022011099A1 (en) 2020-07-08 2022-01-13 Regents Of The University Of Minnesota Modified hexosaminidase and uses thereof
AU2021308681A1 (en) 2020-07-16 2023-03-09 Acuitas Therapeutics, Inc. Cationic lipids for use in lipid nanoparticles
JP2023549780A (ja) 2020-11-04 2023-11-29 ジュノー セラピューティクス インコーポレイテッド 改変されたインバリアントcd3免疫グロブリンスーパーファミリー鎖遺伝子座からキメラ受容体を発現する細胞ならびに関連するポリヌクレオチドおよび方法
AR125199A1 (es) 2021-03-23 2023-06-21 Iovance Biotherapeutics Inc Edición génica cish de linfocitos infiltrantes de tumores y usos de los mismos en inmunoterapia
WO2023035011A1 (en) 2021-09-03 2023-03-09 North Carolina State University Compositions and methods for conferring resistance to geminivirus
US20230141563A1 (en) 2021-10-12 2023-05-11 Selecta Biosciences, Inc. Methods and compositions for attenuating anti-viral transfer vector igm responses
US20230149563A1 (en) 2021-10-27 2023-05-18 Regeneron Pharmaceuticals, Inc. Compositions and methods for expressing factor ix for hemophilia b therapy
EP4426832A1 (en) 2021-11-03 2024-09-11 The J. David Gladstone Institutes, A Testamentary Trust Established under The Will of J. David Gladstone Precise genome editing using retrons
WO2023081900A1 (en) 2021-11-08 2023-05-11 Juno Therapeutics, Inc. Engineered t cells expressing a recombinant t cell receptor (tcr) and related systems and methods
IL312971A (en) 2021-12-08 2024-07-01 Regeneron Pharma Mutant myocilin disease model and uses thereof
KR20240123832A (ko) 2021-12-16 2024-08-14 아퀴타스 테라퓨틱스 인크. 지질 나노입자 제형에 사용하기 위한 지질
WO2023122805A1 (en) 2021-12-20 2023-06-29 Vestaron Corporation Sorbitol driven selection pressure method
WO2023141602A2 (en) 2022-01-21 2023-07-27 Renagade Therapeutics Management Inc. Engineered retrons and methods of use
US20250032642A1 (en) 2022-02-02 2025-01-30 Regeneron Pharmaceuticals, Inc. Crispr-mediated transgene insertion in neonatal cells
WO2023150393A2 (en) 2022-02-07 2023-08-10 Ensoma, Inc. Inhibitor-resistant mgmt modifications and modification of mgmt-encoding nucleic acids
WO2023150798A1 (en) 2022-02-07 2023-08-10 Regeneron Pharmaceuticals, Inc. Compositions and methods for defining optimal treatment timeframes in lysosomal disease
EP4476245A1 (en) 2022-02-11 2024-12-18 Regeneron Pharmaceuticals, Inc. Compositions and methods for screening 4r tau targeting agents
US20230357437A1 (en) 2022-03-09 2023-11-09 Selecta Biosciences, Inc. Immunosuppressants in combination with anti-igm agents and related dosing
WO2023220043A1 (en) 2022-05-09 2023-11-16 Synteny Therapeutics, Inc. Erythroparvovirus with a modified genome for gene therapy
EP4522201A1 (en) 2022-05-09 2025-03-19 Synteny Therapeutics, Inc. Erythroparvovirus compositions and methods for gene therapy
US20250295810A1 (en) 2022-05-09 2025-09-25 Synteny Therapeutics, Inc. Erythroparvovirus with a modified capsid for gene therapy
WO2024026474A1 (en) 2022-07-29 2024-02-01 Regeneron Pharmaceuticals, Inc. Compositions and methods for transferrin receptor (tfr)-mediated delivery to the brain and muscle
WO2024044723A1 (en) 2022-08-25 2024-02-29 Renagade Therapeutics Management Inc. Engineered retrons and methods of use
CA3265590A1 (en) 2022-09-28 2024-04-04 Regeneron Pharmaceuticals, Inc. MODIFIED ANTIBODY-RESISTANT RECEPTORS TO IMPROVE CELL-BASED THERAPIES
US20240182561A1 (en) 2022-11-04 2024-06-06 Regeneron Pharmaceuticals, Inc. Calcium voltage-gated channel auxiliary subunit gamma 1 (cacng1) binding proteins and cacng1-mediated delivery to skeletal muscle
WO2024100604A1 (en) 2022-11-09 2024-05-16 Juno Therapeutics Gmbh Methods for manufacturing engineered immune cells
AU2023379457A1 (en) 2022-11-14 2025-05-15 Regeneron Pharmaceuticals, Inc. Compositions and methods for fibroblast growth factor receptor 3-mediated delivery to astrocytes
CN121002052A (zh) 2023-02-03 2025-11-21 C3S2 有限公司 用于非病毒生产工程化免疫细胞的方法
WO2024196965A1 (en) 2023-03-23 2024-09-26 Carbon Biosciences, Inc. Parvovirus compositions and related methods for gene therapy
WO2024197242A1 (en) 2023-03-23 2024-09-26 Carbon Biosciences, Inc. Protoparvovirus compositions comprising a protoparvovirus variant vp1 capsid polypeptide and related methods
GB202311501D0 (en) 2023-07-26 2023-09-06 Imperial College Innovations Ltd Zinc finger peptides, encoded nucleic acids, methods and uses
TW202519551A (zh) 2023-07-28 2025-05-16 美商雷傑納榮製藥公司 用於治療酸性神經髓磷脂酶缺乏症之抗TfR:酸性神經髓磷脂酶
TW202513801A (zh) 2023-07-28 2025-04-01 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
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
WO2025049524A1 (en) 2023-08-28 2025-03-06 Regeneron Pharmaceuticals, Inc. Cxcr4 antibody-resistant modified receptors
WO2025049959A2 (en) 2023-09-01 2025-03-06 Renagade Therapeutics Management Inc. Gene editing systems, compositions, and methods for treatment of vexas syndrome
WO2025128981A1 (en) 2023-12-14 2025-06-19 Sohm, Inc. Compositions and methods for genome editing
WO2025132815A1 (en) 2023-12-20 2025-06-26 Novozymes A/S Novel cas nucleases and polynucleotides encoding the same
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
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
US20250345431A1 (en) 2024-05-10 2025-11-13 Juno Therapeutics, Inc. Genetically engineered t cells expressing a cd19 chimeric antigen receptor (car) and uses thereof for allogeneic cell therapy

Family Cites Families (50)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5422251A (en) * 1986-11-26 1995-06-06 Princeton University Triple-stranded nucleic acids
US5176996A (en) * 1988-12-20 1993-01-05 Baylor College Of Medicine Method for making synthetic oligonucleotides which bind specifically to target sites on duplex DNA molecules, by forming a colinear triplex, the synthetic oligonucleotides and methods of use
US5420032A (en) 1991-12-23 1995-05-30 Universitge Laval Homing endonuclease which originates from chlamydomonas eugametos and recognizes and cleaves a 15, 17 or 19 degenerate double stranded nucleotide sequence
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
US5792632A (en) 1992-05-05 1998-08-11 Institut Pasteur Nucleotide sequence encoding the enzyme I-SceI and the uses thereof
US6140466A (en) 1994-01-18 2000-10-31 The Scripps Research Institute Zinc finger protein derivatives and methods therefor
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
US5585245A (en) * 1994-04-22 1996-12-17 California Institute Of Technology Ubiquitin-based split protein sensor
DE69535829D1 (de) 1994-08-20 2008-10-16 Gendaq Ltd Verbesserung in bezug auf bindungsproteine bei der erkennung von 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
US6342345B1 (en) * 1997-04-02 2002-01-29 The Board Of Trustees Of The Leland Stanford Junior University Detection of molecular interactions by reporter subunit complementation
GB9710807D0 (en) 1997-05-23 1997-07-23 Medical Res Council Nucleic acid binding proteins
GB9710809D0 (en) 1997-05-23 1997-07-23 Medical Res Council Nucleic acid binding proteins
EP1291420B1 (en) * 1997-12-05 2009-11-25 Europäisches Laboratorium Für Molekularbiologie (Embl) Novel DNA cloning method relying on the E.coli RECE/RECT recombination system
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
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
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
US7013219B2 (en) 1999-01-12 2006-03-14 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
US20030104526A1 (en) * 1999-03-24 2003-06-05 Qiang Liu Position dependent recognition of GNN nucleotide triplets by zinc fingers
US7030215B2 (en) * 1999-03-24 2006-04-18 Sangamo Biosciences, Inc. Position dependent recognition of GNN nucleotide triplets by zinc fingers
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
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
WO2001088197A2 (en) 2000-05-16 2001-11-22 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
US7947469B2 (en) * 2001-01-22 2011-05-24 Gendaq, Ltd. Modulation of HIV infection
GB0108491D0 (en) 2001-04-04 2001-05-23 Gendaq Ltd Engineering zinc fingers
EP1421177A4 (en) 2001-08-20 2006-06-07 Scripps Research Inst ZINC FINGER FASTENING DOMAINS FOR CNN
WO2003087341A2 (en) 2002-01-23 2003-10-23 The University Of Utah Research Foundation Targeted chromosomal mutagenesis using zinc finger nucleases
EP1504092B2 (en) 2002-03-21 2014-06-25 Sangamo BioSciences, Inc. Methods and compositions for using zinc finger endonucleases to enhance homologous recombination
US7947873B2 (en) * 2002-04-17 2011-05-24 Sangamo Biosciences, Inc. Compositions and methods for regulation of plant gamma-tocopherol methyltransferase
US9447434B2 (en) 2002-09-05 2016-09-20 California Institute Of Technology Use of chimeric nucleases to stimulate gene targeting
AU2004208031B2 (en) * 2003-01-28 2009-10-08 Cellectis Use of meganucleases for inducing homologous recombination ex vivo and in toto in vertebrate somatic tissues and application thereof.
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
AU2004263865B2 (en) * 2003-08-08 2007-05-17 Sangamo Therapeutics, Inc. Methods and compositions for targeted cleavage and recombination
US20070134796A1 (en) * 2005-07-26 2007-06-14 Sangamo Biosciences, Inc. Targeted integration and expression of exogenous nucleic acid sequences
CA2562193A1 (en) * 2004-04-08 2005-10-27 Sangamo Biosciences, Inc. Treatment of neuropathic pain with zinc finger proteins
DK2628794T3 (en) * 2005-10-18 2016-08-15 Prec Biosciences RATIONALE CONSTRUCTED MECHANUCLEAS WITH CHANGED SEQUENCE SPECIFICITY AND DNA BINDING EFFICIENCY
JP5551432B2 (ja) * 2006-05-25 2014-07-16 サンガモ バイオサイエンシーズ, インコーポレイテッド 遺伝子不活性化のための方法と組成物

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