CA2684378C - Targeted integration into the ppp1r12c locus - Google Patents

Targeted integration into the ppp1r12c locus Download PDF

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Publication number
CA2684378C
CA2684378C CA2684378A CA2684378A CA2684378C CA 2684378 C CA2684378 C CA 2684378C CA 2684378 A CA2684378 A CA 2684378A CA 2684378 A CA2684378 A CA 2684378A CA 2684378 C CA2684378 C CA 2684378C
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cell
sequence
seq
domain
exogenous
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CA2684378A1 (en
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Russell Dekelver
Philip D. Gregory
David Paschon
Fyodor Urnov
Phillip Tam
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Sangamo Therapeutics Inc
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Sangamo Biosciences Inc
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    • C12N15/09Recombinant DNA-technology
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90Stable introduction of foreign DNA into chromosome
    • C12N15/902Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
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    • C12N15/10Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/1034Isolating an individual clone by screening libraries
    • C12N15/1082Preparation or screening gene libraries by chromosomal integration of polynucleotide sequences, HR-, site-specific-recombination, transposons, viral vectors
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/16Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22Ribonucleases [RNase]; Deoxyribonucleases [DNase]
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    • C12Y301/00Hydrolases acting on ester bonds (3.1)
    • C12Y301/21Endodeoxyribonucleases producing 5'-phosphomonoesters (3.1.21)
    • C12Y301/21004Type II site-specific deoxyribonuclease (3.1.21.4)
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    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/80Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
    • C07K2319/81Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor containing a Zn-finger domain for DNA binding
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    • C12N2800/00Nucleic acids vectors
    • C12N2800/30Vector systems comprising sequences for excision in presence of a recombinase, e.g. loxP or FRT

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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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CA2684378A 2007-04-26 2008-04-24 Targeted integration into the ppp1r12c locus Active CA2684378C (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US92632207P 2007-04-26 2007-04-26
US60/926,322 2007-04-26
PCT/US2008/005282 WO2008133938A2 (en) 2007-04-26 2008-04-24 Targeted integration into the ppp1r12c locus

Publications (2)

Publication Number Publication Date
CA2684378A1 CA2684378A1 (en) 2008-11-06
CA2684378C true CA2684378C (en) 2016-11-29

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CA2684378A Active CA2684378C (en) 2007-04-26 2008-04-24 Targeted integration into the ppp1r12c locus

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US (5) US8110379B2 (cg-RX-API-DMAC7.html)
EP (1) EP2137310B1 (cg-RX-API-DMAC7.html)
JP (1) JP5400034B2 (cg-RX-API-DMAC7.html)
CN (1) CN101679977B (cg-RX-API-DMAC7.html)
AT (1) ATE489465T1 (cg-RX-API-DMAC7.html)
AU (1) AU2008244473B2 (cg-RX-API-DMAC7.html)
CA (1) CA2684378C (cg-RX-API-DMAC7.html)
DE (1) DE602008003684D1 (cg-RX-API-DMAC7.html)
WO (1) WO2008133938A2 (cg-RX-API-DMAC7.html)

Families Citing this family (158)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2008317354B2 (en) 2007-10-25 2014-04-10 Ospedale San Raffaele S.R.L. Methods and compositions for targeted integration
EP2281050B1 (en) 2008-04-14 2014-04-02 Sangamo BioSciences, Inc. Linear donor constructs for targeted integration
CA2741119C (en) * 2008-10-29 2019-02-12 Sangamo Biosciences, Inc. Methods and compositions for inactivating glutamine synthetase gene expression
WO2010065123A1 (en) 2008-12-04 2010-06-10 Sangamo Biosciences, Inc. Genome editing in rats using zinc-finger nucleases
CA2747124C (en) * 2008-12-17 2018-09-04 Dow Agrosciences Llc Targeted integration into the zp15 locus
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
WO2011007193A1 (en) * 2009-07-17 2011-01-20 Cellectis Viral vectors encoding a dna repair matrix and containing a virion-associated site specific meganuclease for gene targeting
CA2770312A1 (en) 2009-08-11 2011-02-17 Sangamo Biosciences, Inc. Organisms homozygous for targeted modification
US8956828B2 (en) 2009-11-10 2015-02-17 Sangamo Biosciences, Inc. Targeted disruption of T cell receptor genes using engineered zinc finger protein nucleases
MX336846B (es) * 2010-01-22 2016-02-03 Sangamo Biosciences Inc Alteracion genomica dirigida.
AU2011215557B2 (en) 2010-02-09 2016-03-10 Sangamo Therapeutics, Inc. Targeted genomic modification with partially single-stranded donor molecules
WO2011139336A1 (en) 2010-04-26 2011-11-10 Sangamo Biosciences, Inc. Genome editing of a rosa locus using nucleases
KR101974036B1 (ko) 2010-05-03 2019-04-30 상가모 테라퓨틱스, 인코포레이티드 아연 핑거 모듈을 연결하기 위한 조성물
EP2571512B1 (en) 2010-05-17 2017-08-23 Sangamo BioSciences, Inc. Novel dna-binding proteins and uses thereof
US8945868B2 (en) * 2010-07-21 2015-02-03 Sangamo Biosciences, Inc. Methods and compositions for modification of a HLA locus
WO2012047598A1 (en) 2010-09-27 2012-04-12 Sangamo Biosciences, Inc. Methods and compositions for inhibiting viral entry into cells
ES2562421T3 (es) 2010-10-12 2016-03-04 The Children's Hospital Of Philadelphia Métodos y composiciones para tratar la hemofilia B
AU2012286901B2 (en) 2011-07-25 2016-10-27 Sangamo Therapeutics, Inc. Methods and compositions for alteration of a cystic fibrosis transmembrane conductance regulator (CFTR) gene
IL293650B2 (en) 2011-09-21 2024-10-01 Sangamo Therapeutics Inc Methods and compositions for regulation of transgene expression
AU2012328682B2 (en) 2011-10-27 2017-09-21 Sangamo Therapeutics, Inc. Methods and compositions for modification of the HPRT locus
WO2013158309A2 (en) * 2012-04-18 2013-10-24 The Board Of Trustees Of The Leland Stanford Junior University Non-disruptive gene targeting
KR101904508B1 (ko) 2012-04-25 2018-10-04 리제너론 파마슈티칼스 인코포레이티드 큰 표적화 벡터를 사용한 뉴클레아제-중재된 표적화
US10174331B2 (en) 2012-05-07 2019-01-08 Sangamo Therapeutics, Inc. Methods and compositions for nuclease-mediated targeted integration of transgenes
CA2878037C (en) 2012-07-11 2021-08-31 Sangamo Biosciences, Inc. Methods and compositions for delivery of biologics
AU2013289206B2 (en) 2012-07-11 2018-08-09 Sangamo Therapeutics, 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
SG11201500852WA (en) 2012-08-29 2015-04-29 Sangamo Biosciences Inc Methods and compositions for treatment of a genetic condition
CA2886684C (en) 2012-10-10 2023-09-19 Sangamo Biosciences, Inc. T cell modifying compounds and uses thereof
WO2014081855A1 (en) * 2012-11-20 2014-05-30 Universite De Montreal Methods and compositions for muscular dystrophies
EP2929017A4 (en) * 2012-12-05 2016-09-28 Sangamo Biosciences Inc METHOD AND COMPOSITIONS FOR REGULATING METABOLISM DISEASES
BR102013032200A2 (pt) 2012-12-13 2015-11-24 Dow Agrosciences Llc direcionamento preciso de um gene para um locus específico em milho
JP6491113B2 (ja) 2013-02-25 2019-03-27 サンガモ セラピューティクス, インコーポレイテッド ヌクレアーゼ媒介性遺伝子破壊を増強するための方法および組成物
EP2975942B1 (en) 2013-03-21 2018-08-08 Sangamo Therapeutics, Inc. Targeted disruption of t cell receptor genes using engineered zinc finger protein nucleases
KR20200136508A (ko) 2013-04-16 2020-12-07 리제너론 파마슈티칼스 인코포레이티드 랫트 게놈의 표적화된 변형
AU2014262867B2 (en) * 2013-05-10 2019-12-05 Sangamo Therapeutics, Inc. Delivery methods and compositions for nuclease-mediated genome engineering
WO2014186585A2 (en) 2013-05-15 2014-11-20 Sangamo Biosciences, Inc. Methods and compositions for treatment of a genetic condition
CA2920899C (en) 2013-08-28 2023-02-28 Sangamo Biosciences, Inc. Compositions for linking dna-binding domains and cleavage domains
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
WO2015057976A1 (en) 2013-10-17 2015-04-23 Sangamo Biosciences, Inc. Delivery methods and compositions for nuclease-mediated genome engineering in hematopoietic stem cells
MX391904B (es) 2013-11-11 2025-03-12 Sangamo Biosciences Inc Un represor genetico para usarse en el tratamiento de la enfermedad de huntington.
WO2015073683A2 (en) 2013-11-13 2015-05-21 Children's Medical Center Corporation Nuclease-mediated regulation of gene expression
DK3080274T3 (da) 2013-12-09 2020-08-31 Sangamo Therapeutics Inc Fremgangsmåder og sammensætninger til genom-manipulation
JP6174811B2 (ja) 2013-12-11 2017-08-02 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. ゲノムの標的改変のための方法及び組成物
DK3102673T3 (da) 2014-02-03 2020-07-06 Sangamo Therapeutics Inc Fremgangsmåder og sammensætninger til behandling af beta-talassæmi
KR20150096064A (ko) * 2014-02-14 2015-08-24 서울대학교산학협력단 Mdck ⅱ 세포의 크로모좀에서 aavs1 위치를 인식하고 이곳에 외래 유전자를 도입하여 안정하게 발현시키는 방법
CA2939942A1 (en) 2014-02-24 2015-08-27 Sangamo Biosciences, Inc. Methods and compositions for nuclease-mediated targeted integration
WO2015143046A2 (en) 2014-03-18 2015-09-24 Sangamo Biosciences, Inc. Methods and compositions for regulation of zinc finger protein expression
CA2939847C (en) 2014-03-21 2023-09-05 The Board Of Trustees Of The Leland Stanford Junior University Genome editing without nucleases
US9522936B2 (en) 2014-04-24 2016-12-20 Sangamo Biosciences, Inc. Engineered transcription activator like effector (TALE) proteins
US20170037431A1 (en) * 2014-05-01 2017-02-09 University Of Washington In vivo Gene Engineering with Adenoviral Vectors
GB201407852D0 (en) 2014-05-02 2014-06-18 Iontas Ltd Preparation of libraries od protein variants expressed in eukaryotic cells and use for selecting binding molecules
BR112016025849A2 (pt) 2014-05-08 2017-10-17 Chdi Foundation Inc métodos e composições para o tratamento da doença de huntington
US9574211B2 (en) 2014-05-13 2017-02-21 Sangamo Biosciences, Inc. Methods and compositions for prevention or treatment of a disease
WO2015188056A1 (en) 2014-06-05 2015-12-10 Sangamo Biosciences, Inc. Methods and compositions for nuclease design
RU2704283C9 (ru) 2014-06-06 2020-02-07 Регенерон Фармасьютикалз, Инк. Способы и композиции для модификации целевого локуса
PT3161128T (pt) 2014-06-26 2018-11-21 Regeneron Pharma Métodos e composições para modificações genéticas alvejadas e métodos de utilização
WO2016014794A1 (en) 2014-07-25 2016-01-28 Sangamo Biosciences, Inc. Methods and compositions for modulating nuclease-mediated genome engineering in hematopoietic stem cells
WO2016014837A1 (en) 2014-07-25 2016-01-28 Sangamo Biosciences, Inc. Gene editing for hiv gene therapy
WO2016019144A2 (en) 2014-07-30 2016-02-04 Sangamo Biosciences, Inc. Gene correction of scid-related genes in hematopoietic stem and progenitor cells
CN113699113A (zh) 2014-09-16 2021-11-26 桑格摩治疗股份有限公司 用于造血干细胞中核酸酶介导的基因组工程化和校正的方法和组合物
EP3207124B1 (en) 2014-10-15 2019-06-26 Regeneron Pharmaceuticals, Inc. Methods and compositions for generating or maintaining pluripotent cells
EP3521437B1 (en) 2014-11-21 2025-01-15 Regeneron Pharmaceuticals, Inc. Methods for targeted genetic modification using paired guide rnas
US10889834B2 (en) 2014-12-15 2021-01-12 Sangamo Therapeutics, Inc. Methods and compositions for enhancing targeted transgene integration
SG11201704646YA (en) 2014-12-19 2017-07-28 Regeneron Pharma Methods and compositions for targeted genetic modification through single-step multiple targeting
US20180002379A1 (en) 2015-01-21 2018-01-04 Sangamo Therapeutics, Inc. Methods and compositions for identification of highly specific nucleases
US20180022781A1 (en) 2015-02-13 2018-01-25 Inserm (Institut National De La Sante Et De La Recherche Medicale) Polypeptides for engineering integrase chimeric proteins and their use in gene therapy
US10179918B2 (en) 2015-05-07 2019-01-15 Sangamo Therapeutics, Inc. Methods and compositions for increasing transgene activity
MX382223B (es) 2015-05-12 2025-03-13 Sangamo Therapeutics Inc Regulacion de expresion genica mediada por nucleasa.
US9957501B2 (en) 2015-06-18 2018-05-01 Sangamo Therapeutics, Inc. Nuclease-mediated regulation of gene expression
CA2991301A1 (en) 2015-07-13 2017-01-19 Sangamo Therapeutics, Inc. Delivery methods and compositions for nuclease-mediated genome engineering
JP6713691B2 (ja) * 2015-08-20 2020-07-01 大学共同利用機関法人情報・システム研究機構 動物細胞ゲノム部位特異的外来dna挿入方法及び前記挿入方法を用いて得られる細胞
RS66924B1 (sr) 2015-09-23 2025-07-31 Sangamo Therapeutics Inc Htt represori i njihova upotreba
AU2016343887B2 (en) 2015-10-28 2023-04-06 Sangamo Therapeutics, Inc. Liver-specific constructs, factor VIII expression cassettes and methods of use thereof
AU2016361350B2 (en) 2015-11-23 2023-04-06 Sangamo Therapeutics, Inc. Methods and compositions for engineering immunity
EP3389677B1 (en) 2015-12-18 2024-06-26 Sangamo Therapeutics, Inc. Targeted disruption of the t cell receptor
AU2016374253B2 (en) 2015-12-18 2021-10-21 Sangamo Therapeutics, Inc. Targeted disruption of the MHC cell receptor
CA3010738A1 (en) 2016-01-15 2017-07-20 Sangamo Therapeutics, Inc. Methods and compositions for the treatment of neurologic disease
CA3011049A1 (en) 2016-02-02 2017-08-10 Sangamo Therapeutics, Inc. Compositions for linking dna-binding domains and cleavage domains
CN106148286B (zh) * 2016-06-29 2019-10-29 牛刚 一种用于检测热原的细胞模型的构建方法和细胞模型及热原检测试剂盒
CA3034882A1 (en) 2016-08-24 2018-03-01 Sangamo Therapeutics, Inc. Regulation of gene expression using engineered nucleases
BR112019003327A2 (pt) 2016-08-24 2019-07-02 Sangamo Therapeutics, Inc. nucleases manipuladas de alvo específico
EP3509645A4 (en) 2016-09-07 2020-06-17 Sangamo Therapeutics, Inc. MODULATION OF LIVER GENES
EP3528852B1 (en) 2016-10-20 2025-09-03 Sangamo Therapeutics, Inc. Methods and compositions for the treatment of fabry disease
AU2017347928A1 (en) 2016-10-31 2019-05-09 Sangamo Therapeutics, Inc. Gene correction of scid-related genes in hematopoietic stem and progenitor cells
AU2018219895C1 (en) * 2017-02-09 2024-10-03 Indapta Therapeutics, Inc. Engineered Natural Killer (NK) cells and compositions and methods thereof
CN106978438B (zh) * 2017-02-27 2020-08-28 北京大北农生物技术有限公司 提高同源重组效率的方法
EP4410317A3 (en) 2017-04-28 2024-10-30 Acuitas Therapeutics Inc. Novel carbonyl lipids and lipid nanoparticle formulations for delivery of nucleic acids
JP2020518245A (ja) 2017-05-03 2020-06-25 サンガモ セラピューティクス, インコーポレイテッド 嚢胞性線維症膜貫通コンダクタンス制御因子(cftr)遺伝子の改変のための方法および組成物
US11512287B2 (en) 2017-06-16 2022-11-29 Sangamo Therapeutics, Inc. Targeted disruption of T cell and/or HLA receptors
US20190032156A1 (en) 2017-07-31 2019-01-31 Regeneron Pharmaceuticals, Inc. Methods and compositions for assessing crispr/cas-induced recombination with an exogenous donor nucleic acid in vivo
BR112020001364A2 (pt) 2017-07-31 2020-08-11 Regeneron Pharmaceuticals, Inc. métodos para testar e modificar a capacidade de uma crispr/cas nuclease.
WO2019028023A2 (en) 2017-07-31 2019-02-07 Regeneron Pharmaceuticals, Inc. METHODS AND COMPOSITIONS FOR EVALUATING CRISPR / CAS MEDIATED DISRUPTION OR EXCISION AND CRISPR / CAS INDUCED RECOMBINATION USING IN VIVO EXOGENIC DONOR NUCLEIC ACID
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
CA3089587A1 (en) 2018-02-08 2019-08-15 Sangamo Therapeutics, Inc. Engineered target specific nucleases
CN112041432A (zh) 2018-02-15 2020-12-04 纪念斯隆-凯特林癌症中心 Foxp3靶向剂组合物以及用于过继细胞疗法的使用方法
WO2019183123A1 (en) 2018-03-19 2019-09-26 Regeneron Pharmaceuticals, Inc. Transcription modulation in animals using crispr/cas systems
CN112585276A (zh) 2018-04-05 2021-03-30 朱诺治疗学股份有限公司 产生表达重组受体的细胞的方法和相关组合物
US20210015869A1 (en) 2018-04-05 2021-01-21 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)
US11690921B2 (en) 2018-05-18 2023-07-04 Sangamo Therapeutics, Inc. Delivery of target specific nucleases
CN112867792A (zh) 2018-08-23 2021-05-28 桑格摩生物治疗股份有限公司 工程化靶特异性碱基编辑器
EP4268831A3 (en) 2018-09-12 2024-05-22 Fred Hutchinson Cancer Center Reducing cd33 expression to selectively protect therapeutic cells
KR20210060533A (ko) 2018-09-18 2021-05-26 상가모 테라퓨틱스, 인코포레이티드 프로그램화된 세포 사멸 1 (pd1) 특이적 뉴클레아제
IL322436A (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
EP3867380A2 (en) 2018-10-18 2021-08-25 Intellia Therapeutics, Inc. Compositions and methods for expressing factor ix
TW202028461A (zh) 2018-10-18 2020-08-01 美商英特利亞醫療公司 核酸構築體及使用方法
KR20210123289A (ko) 2018-11-21 2021-10-13 인답타 세라뷰틱스 인코포레이티드 자연 살해 세포 서브세트의 증식을 위한 방법 및 관련 조성물 및 방법
KR20210094609A (ko) 2018-11-28 2021-07-29 포티 세븐, 인코포레이티드 절제 레짐에 대해 저항성인 유전적으로 변형된 hspc
US11453639B2 (en) 2019-01-11 2022-09-27 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
BR112021019512A2 (pt) 2019-04-03 2022-02-15 Regeneron Pharma Métodos para inserir uma sequência de codificação de proteína de ligação ao antígeno e para tratar ou efetuar a profilaxia de uma doença em um animal, animal, célula, genoma, ácido nucleico doador exógeno, gene de porto seguro, e, agente de nuclease ou um ou mais ácidos nucleicos
EP3962519A1 (en) 2019-05-01 2022-03-09 Juno Therapeutics, Inc. Cells expressing a chimeric receptor from a modified cd247 locus, related polynucleotides and methods
KR20220016475A (ko) 2019-05-01 2022-02-09 주노 쎄러퓨티크스 인코퍼레이티드 변형된 tgfbr2 유전자 좌에서 재조합 수용체를 발현하는 세포, 관련 폴리뉴클레오티드 및 방법
BR112021026832A2 (pt) 2019-07-02 2022-05-10 Hutchinson Fred Cancer Res Vetores ad35 recombinantes e aprimoramentos da terapia gênica relacionada
WO2021072115A1 (en) 2019-10-08 2021-04-15 Regents Of The University Of Minnesota Crispr-mediated human genome editing with vectors
AU2020379046B2 (en) 2019-11-08 2025-03-13 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
KR20230022868A (ko) 2020-05-13 2023-02-16 주노 쎄러퓨티크스 인코퍼레이티드 재조합 수용체를 발현하는 공여자-배치 세포의 제조 방법
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
EP4153739A1 (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
WO2021260186A1 (en) 2020-06-26 2021-12-30 Juno Therapeutics Gmbh Engineered t cells conditionally expressing a recombinant receptor, related polynucleotides and methods
WO2022011099A1 (en) 2020-07-08 2022-01-13 Regents Of The University Of Minnesota Modified hexosaminidase and uses thereof
FI4182297T3 (fi) 2020-07-16 2025-12-05 Acuitas Therapeutics Inc Kationisia lipidejä käytettäväksi lipidinanohiukkasissa
US20230398148A1 (en) 2020-11-04 2023-12-14 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
MX2024005124A (es) 2021-10-27 2024-05-16 Regeneron Pharma Composiciones y metodos para expresar el factor ix para terapia para la hemofilia b.
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
WO2023108047A1 (en) 2021-12-08 2023-06-15 Regeneron Pharmaceuticals, Inc. Mutant myocilin disease model and uses thereof
US12129223B2 (en) 2021-12-16 2024-10-29 Acuitas Therapeutics, Inc. Lipids for use in lipid nanoparticle formulations
WO2023150623A2 (en) 2022-02-02 2023-08-10 Regeneron Pharmaceuticals, Inc. Anti-tfr:gaa and anti-cd63:gaa insertion for treatment of pompe disease
WO2023150798A1 (en) 2022-02-07 2023-08-10 Regeneron Pharmaceuticals, Inc. Compositions and methods for defining optimal treatment timeframes in lysosomal disease
EP4476331A2 (en) 2022-02-07 2024-12-18 Ensoma, Inc. Inhibitor-resistant mgmt modifications and modification of mgmt-encoding nucleic acids
EP4522210A1 (en) 2022-05-09 2025-03-19 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
WO2023220040A1 (en) 2022-05-09 2023-11-16 Synteny Therapeutics, Inc. Erythroparvovirus with a modified capsid for gene therapy
EP4561636A1 (en) 2022-07-29 2025-06-04 Regeneron Pharmaceuticals, Inc. Compositions and methods for transferrin receptor (tfr)-mediated delivery to the brain and muscle
WO2024073606A1 (en) 2022-09-28 2024-04-04 Regeneron Pharmaceuticals, Inc. Antibody resistant modified receptors to enhance 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
EP4615960A1 (en) 2022-11-09 2025-09-17 C3S2 GmbH Methods for manufacturing engineered immune cells
EP4619438A2 (en) 2022-11-14 2025-09-24 Regeneron Pharmaceuticals, Inc. Compositions and methods for fibroblast growth factor receptor 3-mediated delivery to astrocytes
EP4658675A1 (en) 2023-02-03 2025-12-10 C3S2 GmbH Methods for non-viral manufacturing of engineered immune cells
WO2024168253A1 (en) 2023-02-10 2024-08-15 Possible Medicines Llc Delivery of an rna guided recombination system
WO2024168265A1 (en) 2023-02-10 2024-08-15 Possible Medicines Llc Aav delivery of rna guided recombination system
WO2024197242A1 (en) 2023-03-23 2024-09-26 Carbon Biosciences, Inc. Protoparvovirus compositions comprising a protoparvovirus variant vp1 capsid polypeptide and related methods
WO2024196965A1 (en) 2023-03-23 2024-09-26 Carbon Biosciences, Inc. Parvovirus compositions and related methods for gene therapy
AR133385A1 (es) 2023-07-28 2025-09-24 Regeneron Pharma Inserción anti-tfr:gaa y anti-cd63:gaa para el tratamiento de la enfermedad de pompe
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
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
WO2025132815A1 (en) 2023-12-20 2025-06-26 Novozymes A/S Novel cas nucleases and polynucleotides encoding the same
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 (37)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5487994A (en) 1992-04-03 1996-01-30 The Johns Hopkins University Insertion and deletion mutants of FokI restriction endonuclease
US5436150A (en) 1992-04-03 1995-07-25 The Johns Hopkins University Functional domains in flavobacterium okeanokoities (foki) restriction endonuclease
US5356802A (en) 1992-04-03 1994-10-18 The Johns Hopkins University Functional domains in flavobacterium okeanokoites (FokI) restriction endonuclease
AU704601B2 (en) 1994-01-18 1999-04-29 Scripps Research Institute, The 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
US6242568B1 (en) 1994-01-18 2001-06-05 The Scripps Research Institute Zinc finger protein derivatives and methods therefor
USRE39229E1 (en) 1994-08-20 2006-08-08 Gendaq Limited Binding proteins for recognition of DNA
GB9824544D0 (en) 1998-11-09 1999-01-06 Medical Res Council Screening system
US5789538A (en) 1995-02-03 1998-08-04 Massachusetts Institute Of Technology Zinc finger proteins with high affinity new DNA binding specificities
US5925523A (en) 1996-08-23 1999-07-20 President & Fellows Of Harvard College Intraction trap assay, reagents and uses thereof
GB2338237B (en) 1997-02-18 2001-02-28 Actinova Ltd In vitro peptide or protein expression library
GB9703369D0 (en) 1997-02-18 1997-04-09 Lindqvist Bjorn H Process
GB9710809D0 (en) 1997-05-23 1997-07-23 Medical Res Council Nucleic acid binding proteins
GB9710807D0 (en) 1997-05-23 1997-07-23 Medical Res Council Nucleic acid binding proteins
US6410248B1 (en) 1998-01-30 2002-06-25 Massachusetts Institute Of Technology General strategy for selecting high-affinity zinc finger proteins for diverse DNA target sites
US6479626B1 (en) 1998-03-02 2002-11-12 Massachusetts Institute Of Technology Poly zinc finger proteins with improved linkers
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
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
US6794136B1 (en) 2000-11-20 2004-09-21 Sangamo Biosciences, Inc. Iterative optimization in the design of binding proteins
AU2001226935B2 (en) 2000-01-24 2006-06-22 Gendaq Limited Nucleic acid binding polypeptides characterized by flexible linkers connected nucleic acid binding modules
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
EP1353941B1 (en) 2001-01-22 2013-03-13 Sangamo BioSciences, Inc. Modified zinc finger binding proteins
GB0108491D0 (en) 2001-04-04 2001-05-23 Gendaq Ltd Engineering zinc fingers
WO2003016496A2 (en) 2001-08-20 2003-02-27 The Scripps Research Institute Zinc finger binding domains for cnn
JP4968498B2 (ja) 2002-01-23 2012-07-04 ユニバーシティ オブ ユタ リサーチ ファウンデーション ジンクフィンガーヌクレアーゼを用いる、標的化された染色体変異誘発
EP2368982A3 (en) 2002-03-21 2011-10-12 Sangamo BioSciences, Inc. Methods and compositions for using zinc finger endonucleases to enhance homologous recombination
US9447434B2 (en) 2002-09-05 2016-09-20 California Institute Of Technology Use of chimeric nucleases to stimulate gene targeting
US7888121B2 (en) 2003-08-08 2011-02-15 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
US8409861B2 (en) 2003-08-08 2013-04-02 Sangamo Biosciences, Inc. Targeted deletion of cellular DNA sequences
CA2534296C (en) * 2003-08-08 2013-03-26 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
CA2579677A1 (en) 2004-09-16 2006-03-30 Sangamo Biosciences, Inc. Compositions and methods for protein production
US20090300782A1 (en) 2005-04-18 2009-12-03 Mount Sinai School Of Medicine Of New York University Targeted gene addition in stem cells
SG10201508995QA (en) 2005-07-26 2015-11-27 Sangamo Biosciences Inc Targeted integration and expression of exogenous nucleic acid sequences
ATE462783T1 (de) 2006-05-25 2010-04-15 Sangamo Biosciences Inc Variante foki-spaltungshälften-domänen

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