JP2009538141A5 - - Google Patents

Download PDF

Info

Publication number
JP2009538141A5
JP2009538141A5 JP2009512175A JP2009512175A JP2009538141A5 JP 2009538141 A5 JP2009538141 A5 JP 2009538141A5 JP 2009512175 A JP2009512175 A JP 2009512175A JP 2009512175 A JP2009512175 A JP 2009512175A JP 2009538141 A5 JP2009538141 A5 JP 2009538141A5
Authority
JP
Japan
Prior art keywords
seq
gene
ccr
zfn
sequence
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
JP2009512175A
Other languages
English (en)
Japanese (ja)
Other versions
JP5551432B2 (ja
JP2009538141A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2007/012588 external-priority patent/WO2007139982A2/en
Publication of JP2009538141A publication Critical patent/JP2009538141A/ja
Publication of JP2009538141A5 publication Critical patent/JP2009538141A5/ja
Application granted granted Critical
Publication of JP5551432B2 publication Critical patent/JP5551432B2/ja
Expired - Fee Related legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Images

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

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2014106579A Division JP5944948B2 (ja) 2006-05-25 2014-05-22 遺伝子不活性化のための方法と組成物

Publications (3)

Publication Number Publication Date
JP2009538141A JP2009538141A (ja) 2009-11-05
JP2009538141A5 true JP2009538141A5 (enExample) 2010-03-04
JP5551432B2 JP5551432B2 (ja) 2014-07-16

Family

ID=38779263

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2009512175A Expired - Fee Related JP5551432B2 (ja) 2006-05-25 2007-05-23 遺伝子不活性化のための方法と組成物
JP2014106579A Expired - Fee Related JP5944948B2 (ja) 2006-05-25 2014-05-22 遺伝子不活性化のための方法と組成物

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2014106579A Expired - Fee Related JP5944948B2 (ja) 2006-05-25 2014-05-22 遺伝子不活性化のための方法と組成物

Country Status (8)

Country Link
US (7) US7951925B2 (enExample)
EP (4) EP2206782A1 (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
WO2007139982A2 (en) * 2006-05-25 2007-12-06 Sangamo Biosciences, Inc. Methods and compositions for gene inactivation
CA2667414C (en) 2006-11-13 2015-12-29 Sangamo Biosciences, Inc. Methods and compositions for modification of the human glucocorticoid receptor locus
EP2155873B1 (en) * 2007-05-23 2016-11-09 Sangamo BioSciences, 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
US8563314B2 (en) 2007-09-27 2013-10-22 Sangamo Biosciences, Inc. Methods and compositions for modulating PD1
US9506120B2 (en) * 2007-09-27 2016-11-29 Sangamo Biosciences, Inc. Rapid in vivo identification of biologically active nucleases
US8936936B2 (en) 2007-10-25 2015-01-20 Sangamo Biosciences, Inc. Methods and compositions for targeted integration
MX2010011511A (es) 2008-04-21 2011-06-09 Danziger Innovations Ltd Vectores de expresion viral de planta y uso de los mismos para generar variaciones genotipicas en genomas de plantas.
EP2297318B1 (en) 2008-05-28 2018-05-23 Sangamo Therapeutics, Inc. Compositions for linking dna-binding domains and cleavage domains
EP2294191B1 (en) 2008-06-10 2016-09-28 Sangamo BioSciences, Inc. Methods and compositions for generation of bax- and bak-deficient cell lines
CA2734235C (en) 2008-08-22 2019-03-26 Sangamo Biosciences, Inc. Methods and compositions for targeted single-stranded cleavage and targeted integration
US8153399B2 (en) * 2008-10-29 2012-04-10 Sangamo Biosciences, Inc. Methods and compositions for inactivating glutamine synthetase gene expression
US20110023150A1 (en) * 2008-12-04 2011-01-27 Sigma-Aldrich Co. Genome editing of genes associated with schizophrenia in animals
US20110030072A1 (en) * 2008-12-04 2011-02-03 Sigma-Aldrich Co. Genome editing of immunodeficiency genes in animals
US20110016539A1 (en) * 2008-12-04 2011-01-20 Sigma-Aldrich Co. Genome editing of neurotransmission-related genes in animals
US20110023145A1 (en) * 2008-12-04 2011-01-27 Sigma-Aldrich Co. Genomic editing of genes involved in autism spectrum disorders
US20110023154A1 (en) * 2008-12-04 2011-01-27 Sigma-Aldrich Co. Silkworm genome editing with zinc finger nucleases
US20110023151A1 (en) * 2008-12-04 2011-01-27 Sigma-Aldrich Co. Genome editing of abc transporters
US20110016541A1 (en) * 2008-12-04 2011-01-20 Sigma-Aldrich Co. Genome editing of sensory-related genes in animals
US20110023153A1 (en) * 2008-12-04 2011-01-27 Sigma-Aldrich Co. Genomic editing of genes involved in alzheimer's disease
US20110023144A1 (en) * 2008-12-04 2011-01-27 Sigma-Aldrich Co. Genomic editing of genes involved in amyotrophyic lateral sclerosis disease
US20110023149A1 (en) * 2008-12-04 2011-01-27 Sigma-Aldrich Co. Genomic editing of genes involved in tumor suppression in animals
US20110023148A1 (en) * 2008-12-04 2011-01-27 Sigma-Aldrich Co. Genome editing of addiction-related genes in animals
US9206404B2 (en) 2008-12-04 2015-12-08 Sangamo Biosciences, Inc. Method of deleting an IgM gene in an isolated rat cell
US20110023147A1 (en) * 2008-12-04 2011-01-27 Sigma-Aldrich Co. Genomic editing of prion disorder-related genes in animals
US20110023158A1 (en) * 2008-12-04 2011-01-27 Sigma-Aldrich Co. Bovine 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
US20110023141A1 (en) * 2008-12-04 2011-01-27 Sigma-Aldrich Co. Genomic editing of genes involved with parkinson's disease
US20110023156A1 (en) * 2008-12-04 2011-01-27 Sigma-Aldrich Co. Feline genome editing with zinc finger nucleases
US20110016543A1 (en) * 2008-12-04 2011-01-20 Sigma-Aldrich Co. Genomic editing of genes involved in inflammation
US20110023139A1 (en) * 2008-12-04 2011-01-27 Sigma-Aldrich Co. Genomic editing of genes involved in cardiovascular disease
US20110016546A1 (en) * 2008-12-04 2011-01-20 Sigma-Aldrich Co. Porcine genome editing with zinc finger nucleases
US20110023146A1 (en) * 2008-12-04 2011-01-27 Sigma-Aldrich Co. Genomic editing of genes involved in secretase-associated disorders
US20110016540A1 (en) * 2008-12-04 2011-01-20 Sigma-Aldrich Co. Genome editing of genes associated with trinucleotide repeat expansion disorders in animals
US20110023143A1 (en) * 2008-12-04 2011-01-27 Sigma-Aldrich Co. Genomic editing of neurodevelopmental genes in animals
US20110023140A1 (en) * 2008-12-04 2011-01-27 Sigma-Aldrich Co. Rabbit genome editing with zinc finger nucleases
WO2010107493A2 (en) 2009-03-20 2010-09-23 Sangamo Biosciences, Inc. Modification of cxcr4 using engineered zinc finger proteins
AU2010235161B2 (en) * 2009-04-09 2015-01-22 Sangamo Therapeutics, Inc. Targeted integration into stem cells
US8772008B2 (en) * 2009-05-18 2014-07-08 Sangamo Biosciences, Inc. Methods and compositions for increasing nuclease activity
EP2449135B1 (en) 2009-06-30 2016-01-06 Sangamo BioSciences, Inc. Rapid screening of biologically active nucleases and isolation of nuclease-modified cells
WO2011016840A2 (en) * 2009-07-28 2011-02-10 Sangamo Biosciences, Inc. Methods and compositions for treating trinucleotide repeat disorders
WO2011048600A1 (en) 2009-10-21 2011-04-28 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
EP2504429B1 (en) * 2009-11-27 2016-04-20 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
JP5902631B2 (ja) * 2010-01-22 2016-04-13 ダウ アグロサイエンシィズ エルエルシー 標的化ゲノム変更
CA2788560A1 (en) 2010-02-08 2011-08-11 Sangamo Biosciences, Inc. Engineered cleavage half-domains
WO2011100058A1 (en) 2010-02-09 2011-08-18 Sangamo Biosciences, Inc. Targeted genomic modification with partially single-stranded donor molecules
EP2563918B1 (en) 2010-04-26 2016-11-23 Sangamo BioSciences, Inc. Genome editing of a rosa locus using zinc-finger nucleases
CN102959087B (zh) 2010-05-03 2015-04-29 桑格摩生物科学股份有限公司 用于连接锌指模块的组合物
CN103025344B (zh) 2010-05-17 2016-06-29 桑格摩生物科学股份有限公司 新型dna-结合蛋白及其用途
EP2580331A4 (en) * 2010-06-14 2013-11-27 Univ Iowa State Res Found Inc NUCLEASE ACTIVITY OF THE TAL EFFECTOR AND FUSION PROTEIN FOKI
US8945868B2 (en) 2010-07-21 2015-02-03 Sangamo Biosciences, Inc. Methods and compositions for modification of a HLA locus
KR20180121665A (ko) * 2010-07-23 2018-11-07 시그마-알드리치 컴퍼니., 엘엘씨 표적화 엔도뉴클레아제 및 단일-가닥 핵산을 사용하는 게놈 편집
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
CA2811364C (en) 2010-09-27 2022-01-04 Sangamo Biosciences, Inc. Methods and compositions for inhibiting viral entry into cells
JP6018069B2 (ja) 2010-10-12 2016-11-02 ザ・チルドレンズ・ホスピタル・オブ・フィラデルフィアThe Children’S Hospital Of Philadelphia 血友病bを治療する方法及び組成物
EP2441774B1 (en) 2010-10-15 2018-12-12 Fundació Centre de Regulació Genòmica Peptides having zinc finger domains and uses thereof
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
EP3498833B1 (en) 2011-09-21 2023-08-16 Sangamo Therapeutics, Inc. Methods and compositions for regulation of transgene expression
WO2013043638A1 (en) * 2011-09-23 2013-03-28 Iowa State University Research Foundation, Inc. Monomer architecture of tal nuclease or zinc finger nuclease for dna modification
CA2852955C (en) 2011-10-27 2021-02-16 Sangamo Biosciences, 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
MX374399B (es) 2012-02-29 2025-03-06 Sangamo Biosciences Inc Composiciones y sus usos para tratar y prevenir la enfermedad de huntington.
CA2871524C (en) 2012-05-07 2021-07-27 Sangamo Biosciences, Inc. Methods and compositions for nuclease-mediated targeted integration of transgenes
SI3401400T1 (sl) 2012-05-25 2019-10-30 Univ California Postopki in sestavki za RNA usmerjeno modifikacijo tarčne DNA in za RNA usmerjeno modulacijo prepisovanja
CA2875618C (en) 2012-07-11 2021-04-27 Sangamo Biosciences, Inc. Methods and compositions for the treatment of lysosomal storage diseases
US10648001B2 (en) 2012-07-11 2020-05-12 Sangamo Therapeutics, Inc. Method of treating mucopolysaccharidosis type I or II
US10883119B2 (en) 2012-07-11 2021-01-05 Sangamo Therapeutics, Inc. Methods and compositions for delivery of biologics
US20150104873A1 (en) * 2012-07-11 2015-04-16 University of Nevada, Las Vegas Genome Surgery with Paired, Permeant Endonuclease Construct
SG11201500852WA (en) 2012-08-29 2015-04-29 Sangamo Biosciences Inc Methods and compositions for treatment of a genetic condition
EP2906684B8 (en) 2012-10-10 2020-09-02 Sangamo Therapeutics, Inc. T cell modifying compounds and uses thereof
US20140140969A1 (en) * 2012-11-20 2014-05-22 Sangamo Biosciences, Inc. Methods and compositions for muscular dystrophies
EP2929017A4 (en) 2012-12-05 2016-09-28 Sangamo Biosciences Inc METHOD AND COMPOSITIONS FOR REGULATING METABOLISM DISEASES
EP3138911B1 (en) 2012-12-06 2018-12-05 Sigma Aldrich Co. LLC Crispr-based genome modification and regulation
EP2958996B1 (en) 2013-02-25 2019-10-16 Sangamo Therapeutics, Inc. Methods and compositions for enhancing nuclease-mediated gene disruption
US20140315985A1 (en) 2013-03-14 2014-10-23 Caribou Biosciences, Inc. Compositions and methods of nucleic acid-targeting nucleic acids
AU2014235968B2 (en) 2013-03-21 2018-05-24 Ospedale San Raffaele Srl Targeted disruption of T cell receptor genes using engineered zinc finger protein nucleases
JP2016522679A (ja) * 2013-04-04 2016-08-04 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 CRISPR/Cas系を用いたゲノム編集の治療的使用
JP2016518142A (ja) * 2013-05-10 2016-06-23 サンガモ バイオサイエンシーズ, インコーポレイテッド ヌクレアーゼ媒介ゲノム遺伝子操作のための送達方法および組成物
HK1223401A1 (zh) 2013-05-15 2017-07-28 桑格摩生物科学股份有限公司 用於治疗遗传病状的方法和组合物
JP6588438B2 (ja) 2013-08-28 2019-10-09 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
EP3057432B1 (en) 2013-10-17 2018-11-21 Sangamo Therapeutics, Inc. Delivery methods and compositions for nuclease-mediated genome engineering in hematopoietic stem cells
CN110713995B (zh) * 2013-10-17 2023-08-01 桑格摩生物科学股份有限公司 用于核酸酶介导的基因组工程改造的递送方法和组合物
EP3068905A4 (en) 2013-11-11 2017-07-05 Sangamo BioSciences, Inc. Methods and compositions for treating huntington's disease
ES2895069T3 (es) 2013-11-13 2022-02-17 Childrens Medical Center Regulación de la expresión génica mediada por nucleasa
AU2014350051A1 (en) * 2013-11-18 2016-07-07 Crispr Therapeutics Ag CRISPR-Cas system materials and methods
CA3229275A1 (en) 2013-12-09 2015-06-18 Sangamo Biosciences, Inc. Methods and compositions for genome engineering
DK3102673T3 (da) 2014-02-03 2020-07-06 Sangamo Therapeutics Inc Fremgangsmåder og sammensætninger til behandling af beta-talassæmi
CA2939942A1 (en) 2014-02-24 2015-08-27 Sangamo Biosciences, Inc. Methods and compositions for nuclease-mediated targeted integration
KR101576728B1 (ko) * 2014-03-06 2015-12-10 가톨릭대학교 산학협력단 면역세포 백신 및 이의 용도
EP3119878B1 (en) 2014-03-18 2021-05-26 Sangamo Therapeutics, 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
AU2015255877B2 (en) 2014-05-08 2020-03-26 Chdi Foundation, Inc. Methods and compositions for treating huntington's disease
WO2015175642A2 (en) 2014-05-13 2015-11-19 Sangamo Biosciences, Inc. Methods and compositions for prevention or treatment of a disease
EP3152319A4 (en) 2014-06-05 2017-12-27 Sangamo BioSciences, Inc. Methods and compositions for nuclease design
WO2016014837A1 (en) 2014-07-25 2016-01-28 Sangamo Biosciences, Inc. Gene editing for hiv gene therapy
US9816074B2 (en) 2014-07-25 2017-11-14 Sangamo Therapeutics, Inc. Methods and compositions for modulating nuclease-mediated genome engineering in hematopoietic stem cells
WO2016019144A2 (en) 2014-07-30 2016-02-04 Sangamo Biosciences, Inc. Gene correction of scid-related genes in hematopoietic stem and progenitor cells
IL292575A (en) 2014-09-07 2022-06-01 Selecta Biosciences Inc Methods and preparations for weakening immune responses by modulating gene expression in an antiviral transfer system
AU2015317793B2 (en) 2014-09-16 2021-11-11 Sangamo Therapeutics, Inc. Methods and compositions for nuclease-mediated genome engineering and correction in hematopoietic stem cells
KR20170096998A (ko) 2014-11-05 2017-08-25 보이저 테라퓨틱스, 인크. 파킨슨병의 치료를 위한 aadc 폴리뉴클레오티드
WO2016077689A1 (en) 2014-11-14 2016-05-19 Voyager Therapeutics, Inc. Modulatory polynucleotides
WO2016077687A1 (en) 2014-11-14 2016-05-19 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
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
US20180002379A1 (en) 2015-01-21 2018-01-04 Sangamo Therapeutics, Inc. Methods and compositions for identification of highly specific nucleases
WO2016128549A1 (en) 2015-02-13 2016-08-18 INSERM (Institut National de la Santé et de la Recherche Médicale) Polypeptides for engineering integrase chimeric proteins and their use in gene therapy
EP4600366A3 (en) 2015-03-31 2025-10-22 SOHM, Inc. Cas 9 retroviral integrase systems for targeted incorporation of a dna sequence into a genome of a cell
AU2016243052C1 (en) * 2015-04-03 2022-11-24 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
EP3294342A4 (en) 2015-05-08 2018-11-07 President and Fellows of Harvard College Universal donor stem cells and related methods
BR112017024115A2 (pt) * 2015-05-12 2018-08-07 Sangamo Therapeutics Inc regulação de expressão genética mediada por nuclease
CN107614680A (zh) * 2015-05-14 2018-01-19 南加利福尼亚大学 利用重组核酸内切酶系统的最佳化基因编辑
US9957501B2 (en) 2015-06-18 2018-05-01 Sangamo Therapeutics, Inc. Nuclease-mediated regulation of gene expression
EP3322297B1 (en) 2015-07-13 2024-12-04 Sangamo Therapeutics, Inc. Delivery methods and compositions for nuclease-mediated genome engineering
US10435441B2 (en) 2015-09-23 2019-10-08 Sangamo Therapeutics, Inc. HTT repressors and uses thereof
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
CN115141836B (zh) 2015-10-28 2024-08-27 桑格摩生物治疗股份有限公司 肝特异性构建体、因子viii表达盒及其使用方法
WO2017079428A1 (en) 2015-11-04 2017-05-11 President And Fellows Of Harvard College Site specific germline modification
JP6976249B2 (ja) 2015-11-23 2021-12-08 サンガモ セラピューティクス, インコーポレイテッド 免疫を工学操作するための方法および組成物
JP2018537112A (ja) 2015-12-18 2018-12-20 サンガモ セラピューティクス, インコーポレイテッド Mhc細胞受容体の標的化破壊
AU2016369490C1 (en) 2015-12-18 2021-12-23 Sangamo Therapeutics, Inc. Targeted disruption of the T cell receptor
WO2017123757A1 (en) 2016-01-15 2017-07-20 Sangamo Therapeutics, Inc. Methods and compositions for the treatment of neurologic disease
CN109069568B (zh) 2016-02-02 2023-07-07 桑格摩生物治疗股份有限公司 用于连接dna结合结构域和切割结构域的组合物
CA3024448C (en) 2016-05-18 2025-09-09 Voyager Therapeutics, Inc. MODULATING POLYNUCLEOTIDES
BR112018073472A2 (pt) 2016-05-18 2019-08-27 Voyager Therapeutics Inc composições e métodos de tratamento da doença de huntington
IL264792B2 (en) 2016-08-24 2023-10-01 Sangamo Therapeutics Inc Engineered target-specific zinc-finger nucleases
AU2017315406B2 (en) 2016-08-24 2021-04-01 Sangamo Therapeutics, Inc. Regulation of gene expression using engineered nucleases
US10960085B2 (en) 2016-09-07 2021-03-30 Sangamo Therapeutics, Inc. Modulation of liver genes
US11219695B2 (en) 2016-10-20 2022-01-11 Sangamo Therapeutics, Inc. Methods and compositions for the treatment of Fabry disease
EP3532106A4 (en) 2016-10-31 2020-06-24 Sangamo Therapeutics, Inc. GENERAL CORRECTION OF SCID-ASSOCIATED GENES IN HEMOTOPOIETIC STEM CELLS AND PROCUREMENT CELLS
ES2981244T3 (es) 2017-04-28 2024-10-07 Acuitas Therapeutics Inc Novedosas formulaciones de nanopartículas de lípidos de carbonilo y lípidos para la administración de ácidos nucleicos
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
SG11201909868YA (en) 2017-05-05 2019-11-28 Voyager Therapeutics Inc Compositions and methods of treating huntington's disease
WO2018204786A1 (en) 2017-05-05 2018-11-08 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
JP2020533957A (ja) 2017-07-31 2020-11-26 リジェネロン・ファーマシューティカルズ・インコーポレイテッドRegeneron Pharmaceuticals, Inc. Crisprリポーター非ヒト動物およびその使用
WO2019028032A1 (en) 2017-07-31 2019-02-07 Regeneron Pharmaceuticals, Inc. EMBRYONIC STEM CELLS OF TRANSGENIC MOUSE CASES AND MICE AND USES THEREOF
WO2019028029A1 (en) 2017-07-31 2019-02-07 Regeneron Pharmaceuticals, Inc. EVALUATION OF CRISPR / CAS INDUCED RECOMBINATION WITH IN VIVO EXOGENIC DONOR NUCLEIC ACID
AU2018347583A1 (en) 2017-10-13 2020-05-21 Selecta Biosciences, Inc. Methods and compositions for attenuating anti-viral transfer vector IgM responses
CN111565730B (zh) 2017-11-09 2024-09-17 桑格摩生物治疗股份有限公司 细胞因子诱导型含sh2蛋白(cish)基因的遗传修饰
BR112020015488A2 (pt) 2018-02-08 2020-12-08 Sangamo Therapeutics, Inc. Nucleases alvo-específicas modificadas
EP3752601A4 (en) 2018-02-15 2022-03-23 Memorial Sloan-Kettering Cancer Center FOXP3-TARGETING AGENT COMPOSITIONS AND METHODS OF USE FOR ADOPTIVE CELL THERAPY
KR20200139679A (ko) 2018-02-26 2020-12-14 안톨알엑스, 인크. 면역관용성 리포솜 및 그의 사용 방법
CN116349651A (zh) 2018-03-19 2023-06-30 瑞泽恩制药公司 使用crispr/cas系统对动物进行转录调制
MA52656A (fr) 2018-04-05 2021-02-17 Editas Medicine Inc Procédés de production de cellules exprimant un récepteur recombinant et compositions associées
EP3775237A1 (en) 2018-04-05 2021-02-17 Juno Therapeutics, Inc. T cells expressing a recombinant receptor, related polynucleotides and methods
US11421007B2 (en) 2018-04-18 2022-08-23 Sangamo Therapeutics, Inc. Zinc finger protein compositions for modulation of huntingtin (Htt)
JP2021523914A (ja) 2018-05-15 2021-09-09 ボイジャー セラピューティクス インコーポレイテッドVoyager Therapeutics,Inc. パーキンソン病を治療するための組成物および方法
US11690921B2 (en) 2018-05-18 2023-07-04 Sangamo Therapeutics, Inc. Delivery of target specific nucleases
US11834686B2 (en) 2018-08-23 2023-12-05 Sangamo Therapeutics, Inc. Engineered target specific base editors
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
IL281615B1 (en) 2018-09-21 2025-09-01 Acuitas Therapeutics Inc Systems and methods for producing lipid nanoparticles and liposomes
WO2020069029A1 (en) 2018-09-26 2020-04-02 Emendobio Inc. Novel crispr nucleases
SG11202103647YA (en) * 2018-10-11 2021-05-28 Ospedale San Raffaele Srl Selection by means of artificial transactivators
CA3116580A1 (en) 2018-10-18 2020-04-23 Intellia Therapeutics, Inc. Nucleic acid constructs and methods of use
EP3867380A2 (en) 2018-10-18 2021-08-25 Intellia Therapeutics, Inc. Compositions and methods for expressing factor ix
JP2022510634A (ja) 2018-11-28 2022-01-27 フォーティ セブン, インコーポレイテッド 除去レジメンに抵抗性の遺伝的に改変されたhspc
AU2020205717B2 (en) 2019-01-11 2025-09-11 Acuitas Therapeutics, Inc. Lipids for lipid nanoparticle delivery of active agents
WO2020163379A1 (en) 2019-02-05 2020-08-13 Emendobio Inc. Crispr compositions and methods for promoting gene editing of ribosomal protein s19 (rps19) gene
US20220154157A1 (en) 2019-02-06 2022-05-19 Emendobio Inc. New engineered high fidelity cas9
PH12021500032A1 (en) 2019-04-03 2022-05-02 Regeneron Pharma Methods and compositions for insertion of antibody coding sequences into a safe harbor locus
US20220184131A1 (en) 2019-05-01 2022-06-16 Juno Therapeutics, Inc. Cells expressing a recombinant receptor from a modified tgfbr2 locus, related polynucleotides and methods
CN114007640A (zh) 2019-05-01 2022-02-01 朱诺治疗学股份有限公司 从修饰的cd247基因座表达嵌合受体的细胞、相关多核苷酸和方法
AU2020284555A1 (en) 2019-05-28 2021-12-23 Selecta Biosciences, Inc. Methods and compositions for attenuated anti-viral transfer vector immune response
AU2020298572A1 (en) 2019-07-02 2021-11-18 Fred Hutchinson Cancer Center Recombinant Ad35 vectors and related gene therapy improvements
WO2021011882A1 (en) * 2019-07-17 2021-01-21 Case Western Reserve University Long lived t cells for treating hiv infection
WO2021072115A1 (en) 2019-10-08 2021-04-15 Regents Of The University Of Minnesota Crispr-mediated human genome editing with vectors
IL292605B2 (en) 2019-11-08 2025-09-01 Regeneron Pharma CRISPR and AAV strategies for treating childhood X-linked retinoschisis
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
JP2023526278A (ja) 2020-05-13 2023-06-21 ジュノー セラピューティクス インコーポレイテッド 組み換え受容体を発現しているドナーバッチ細胞を産生するための方法
WO2021234388A1 (en) 2020-05-21 2021-11-25 Oxford Genetics Limited Hdr enhancers
GB202007577D0 (en) 2020-05-21 2020-07-08 Oxford Genetics Ltd Hdr enhancers
US20230183751A1 (en) 2020-05-21 2023-06-15 Oxford Genetics Limited Hdr enhancers
GB202007578D0 (en) 2020-05-21 2020-07-08 Univ Oxford Innovation Ltd Hdr enhancers
KR20230042283A (ko) 2020-06-26 2023-03-28 주노 테라퓨틱스 게엠베하 재조합 수용체를 조건부로 발현하는 조작된 t 세포, 관련된 폴리뉴클레오티드 및 방법
GB202010075D0 (en) 2020-07-01 2020-08-12 Imp College Innovations Ltd Therapeutic nucleic acids, peptides and uses
US20230374483A1 (en) 2020-07-08 2023-11-23 Regents Of The University Of Minnesota Modified hexosaminidase and uses thereof
CA3189338A1 (en) 2020-07-16 2022-01-20 Acuitas Therapeutics, Inc. Cationic lipids for use in lipid nanoparticles
EP4240756A1 (en) 2020-11-04 2023-09-13 Juno Therapeutics, Inc. Cells expressing a chimeric receptor from a modified invariant cd3 immunoglobulin superfamily chain locus and related polynucleotides and methods
CN118019546A (zh) 2021-03-23 2024-05-10 艾欧凡斯生物治疗公司 肿瘤浸润淋巴细胞的cish基因编辑及其在免疫疗法中的用途
US12305184B2 (en) 2021-09-03 2025-05-20 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
WO2023077012A1 (en) 2021-10-27 2023-05-04 Regeneron Pharmaceuticals, Inc. Compositions and methods for expressing factor ix for hemophilia b therapy
CA3237482A1 (en) 2021-11-03 2023-05-11 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
CN118632622A (zh) 2021-12-08 2024-09-10 瑞泽恩制药公司 突变型肌纤蛋白疾病模型及其用途
WO2023114943A2 (en) 2021-12-16 2023-06-22 Acuitas Therapeutics, Inc. Lipids for use in lipid nanoparticle formulations
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
EP4473103A2 (en) 2022-02-02 2024-12-11 Regeneron Pharmaceuticals, Inc. Anti-tfr:gaa and anti-cd63:gaa insertions for treatment of pompe disease
US20250135039A1 (en) 2022-02-07 2025-05-01 Ensoma, Inc. Inhibitor-resistant mgmt modifications and modification of mgmt-encoding nucleic acids
EP4475671A1 (en) 2022-02-07 2024-12-18 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
EP4522203A1 (en) 2022-05-09 2025-03-19 Synteny Therapeutics, Inc. Erythroparvovirus with a modified capsid for gene therapy
EP4522201A1 (en) 2022-05-09 2025-03-19 Synteny Therapeutics, Inc. Erythroparvovirus compositions and methods for gene therapy
EP4522210A1 (en) 2022-05-09 2025-03-19 Synteny Therapeutics, Inc. Erythroparvovirus with a modified genome for gene therapy
CN120659627A (zh) 2022-07-29 2025-09-16 瑞泽恩制药公司 用于转铁蛋白受体(tfr)介导的脑和肌肉递送的组合物和方法
WO2024044723A1 (en) 2022-08-25 2024-02-29 Renagade Therapeutics Management Inc. Engineered retrons and methods of use
CN120265314A (zh) 2022-09-28 2025-07-04 瑞泽恩制药公司 抗体抗性修饰受体以增强基于细胞的疗法
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
EP4615960A1 (en) 2022-11-09 2025-09-17 C3S2 GmbH Methods for manufacturing engineered immune cells
JP2025538220A (ja) 2022-11-14 2025-11-26 リジェネロン・ファーマシューティカルズ・インコーポレイテッド アストロサイトへの線維芽細胞増殖因子受容体3媒介送達のための組成物および方法
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
US12383615B2 (en) 2023-03-23 2025-08-12 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
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
WO2025029657A2 (en) 2023-07-28 2025-02-06 Regeneron Pharmaceuticals, Inc. Anti-tfr:gaa and anti-cd63:gaa insertion for treatment of pompe disease
US20250049896A1 (en) 2023-07-28 2025-02-13 Regeneron Pharmaceuticals, Inc. Anti-tfr:acid sphingomyelinase for treatment of acid sphingomyelinase deficiency
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
WO2025235851A1 (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
US5356802A (en) 1992-04-03 1994-10-18 The Johns Hopkins University Functional domains in flavobacterium okeanokoites (FokI) restriction endonuclease
US5436150A (en) 1992-04-03 1995-07-25 The Johns Hopkins University Functional domains in flavobacterium okeanokoities (foki) restriction endonuclease
US5792632A (en) 1992-05-05 1998-08-11 Institut Pasteur Nucleotide sequence encoding the enzyme I-SceI and the uses thereof
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
CA2681922C (en) 1994-01-18 2012-05-15 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
GB9824544D0 (en) 1998-11-09 1999-01-06 Medical Res Council Screening system
JP4118327B2 (ja) * 1994-08-20 2008-07-16 ゲンダック・リミテッド Dna認識のための結合タンパク質におけるまたはそれに関連する改良
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
ES2337027T3 (es) * 1997-12-05 2010-04-20 Europaisches Laboratorium Fur Molekularbiologie (Embl) Nuevo metodo de clonacion de dna basado en el sistema de recombinacion rece-rect de e. coli.
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
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
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
US6599692B1 (en) 1999-09-14 2003-07-29 Sangamo Bioscience, Inc. Functional genomics using zinc finger proteins
US7030215B2 (en) * 1999-03-24 2006-04-18 Sangamo Biosciences, Inc. Position dependent recognition of GNN nucleotide triplets by zinc fingers
US20030104526A1 (en) * 1999-03-24 2003-06-05 Qiang Liu 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
DE60023936T2 (de) 1999-12-06 2006-05-24 Sangamo Biosciences Inc., Richmond Methoden zur verwendung von randomisierten zinkfingerprotein-bibliotheken zur identifizierung von genfunktionen
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
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
WO2002057308A2 (en) 2001-01-22 2002-07-25 Sangamo Biosciences, Inc. Zinc finger polypeptides and their use
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
EP1476547B1 (en) 2002-01-23 2006-12-06 The University of Utah Research Foundation Targeted chromosomal mutagenesis using zinc finger nucleases
AU2003218382B2 (en) 2002-03-21 2007-12-13 Sangamo Therapeutics, Inc. Methods and compositions for using zinc finger endonucleases to enhance homologous recombination
WO2003089452A2 (en) * 2002-04-17 2003-10-30 Sangamo Biosciences, Inc. Compositions and methods for regulation of plant gamma-tocopherol methyltransferase
EP1581610A4 (en) 2002-09-05 2009-05-27 California Inst Of Techn USE OF CHIMERIC NUCLEASES TO STIMULATE GEN-TARGETING
WO2004067753A2 (en) * 2003-01-28 2004-08-12 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
EP3222715A1 (en) * 2003-08-08 2017-09-27 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
AU2005233550B2 (en) * 2004-04-08 2010-11-18 Sangamo Therapeutics, Inc. Treatment of neuropathic pain with zinc finger proteins
WO2007014275A2 (en) * 2005-07-26 2007-02-01 Sangamo Biosciences, Inc. Targeted integration and expression of exogenous nucleic acid sequences
ES2602184T3 (es) * 2005-10-18 2017-02-20 Precision Biosciences Meganucleasas diseñadas racionalmente con especificidad de secuencia y afinidad de unión a ADN alteradas
WO2007139982A2 (en) * 2006-05-25 2007-12-06 Sangamo Biosciences, Inc. Methods and compositions for gene inactivation

Similar Documents

Publication Publication Date Title
JP2009538141A5 (enExample)
ES2575413T3 (es) Cetol-ácido reductoisomerasa que utiliza NADH
Lee et al. Role of nucleotide sequences of loxP spacer region in Cre-mediated recombination
JP2014518641A5 (enExample)
USD562225S1 (en) Support base for solar collector assembly
TR200101093A2 (tr) Mutan DNA polimerazlar kullanılarak, yüksek sıcaklıkta ters transkripsiyon yapılması.
JP2009519705A5 (enExample)
JP2008237210A5 (enExample)
WO2004044210A3 (en) Materials and methods for the efficient production of acetate and other products
WO2008093847A1 (ja) キシリトールデヒドロゲナーゼをコードするdna
JP2005519634A5 (enExample)
JP2003525627A5 (enExample)
TWD119910S1 (zh) 晶舟
FR2885131B1 (fr) Structure cellulaire a base de polymere comprenant des nanotubes de carbone, son procede de preparation et ses applications
JP2001521765A5 (enExample)
JP2005530695A5 (enExample)
EP2299530A4 (en) NOVEL ENZYME ELECTRODE AND FUEL CELL WITH THE ENZYME ELECTRODE
CN102199616B (zh) Hiv-1表型耐药检测载体及其构建方法
USD590767S1 (en) Portable fuel cell system with removable power adaptor
WO2005108622A3 (en) Methods and kits to increase the efficiency of oligonucleotide-directed nucleic acid sequence alteration
KEUN et al. Immobilization of glucose oxidase on multi-wall carbon nanotubes for biofuel cell applications
JP2007513601A5 (enExample)
WO2008131215A3 (en) Engineered phosphite dehydrogenase mutants
Marszalkowski et al. Comparative study on tertiary contacts and folding of RNase P RNAs from a psychrophilic, a mesophilic/radiation-resistant, and a thermophilic bacterium
Damier et al. Molecular cloning