JP5400034B2 - Ｐｐｐ１ｒ１２ｃ座への標的化組込み - Google Patents

Ｐｐｐ１ｒ１２ｃ座への標的化組込み

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Publication number
JP5400034B2
JP5400034B2 JP2010506254A JP2010506254A JP5400034B2 JP 5400034 B2 JP5400034 B2 JP 5400034B2 JP 2010506254 A JP2010506254 A JP 2010506254A JP 2010506254 A JP2010506254 A JP 2010506254A JP 5400034 B2 JP5400034 B2 JP 5400034B2
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cell
sequence
domain
gene
ppp1r12c
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Expired - Fee Related
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JP2010506254A
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Japanese (ja)
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JP2010524500A5 (enExample
JP2010524500A (ja
Inventor
デケルバー，ラッセル
ディー． グレゴリー，フィリップ
パスチョン，デイビッド
ウルノフ，フョードル
タム，フィリップ
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Sangamo Therapeutics Inc
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Sangamo Therapeutics Inc
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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/43504—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from invertebrates
    • C07K14/43595—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from invertebrates from coelenteratae, e.g. medusae
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/1034—Isolating an individual clone by screening libraries
    • C12N15/1082—Preparation or screening gene libraries by chromosomal integration of polynucleotide sequences, HR-, site-specific-recombination, transposons, viral vectors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y301/00—Hydrolases acting on ester bonds (3.1)
    • C12Y301/21—Endodeoxyribonucleases producing 5'-phosphomonoesters (3.1.21)
    • C12Y301/21004—Type II site-specific deoxyribonuclease (3.1.21.4)
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
    • C07K2319/81—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor containing a Zn-finger domain for DNA binding
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/10—Plasmid DNA
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/30—Vector systems comprising sequences for excision in presence of a recombinase, e.g. loxP or FRT

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • Virology (AREA)
  • Bioinformatics & Computational Biology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Mycology (AREA)
  • Cell Biology (AREA)
  • Tropical Medicine & Parasitology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Enzymes And Modification Thereof (AREA)
JP2010506254A 2007-04-26 2008-04-24 Ｐｐｐ１ｒ１２ｃ座への標的化組込み Expired - Fee Related JP5400034B2 (ja)

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 (3)

Publication Number Publication Date
JP2010524500A JP2010524500A (ja) 2010-07-22
JP2010524500A5 JP2010524500A5 (enExample) 2011-05-19
JP5400034B2 true JP5400034B2 (ja) 2014-01-29

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JP2010506254A Expired - Fee Related JP5400034B2 (ja) 2007-04-26 2008-04-24 Ｐｐｐ１ｒ１２ｃ座への標的化組込み

Country Status (9)

Country Link
US (5) US8110379B2 (enExample)
EP (1) EP2137310B1 (enExample)
JP (1) JP5400034B2 (enExample)
CN (1) CN101679977B (enExample)
AT (1) ATE489465T1 (enExample)
AU (1) AU2008244473B2 (enExample)
CA (1) CA2684378C (enExample)
DE (1) DE602008003684D1 (enExample)
WO (1) WO2008133938A2 (enExample)

Families Citing this family (161)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US8936936B2 (en) 2007-10-25 2015-01-20 Sangamo Biosciences, Inc. Methods and compositions for targeted integration
CA2720903C (en) 2008-04-14 2019-01-15 Sangamo Biosciences, Inc. Linear donor constructs for targeted integration
WO2010053518A2 (en) * 2008-10-29 2010-05-14 Sangamo Biosciences, Inc. Methods and compositions for inactivating glutamine synthetase gene expression
ES2627552T3 (es) 2008-12-04 2017-07-28 Sigma Aldrich Company Edición de genoma en ratas usando nucleasas con dedos de cinc
JP2012511926A (ja) * 2008-12-17 2012-05-31 ダウ・アグロサイエンス・エル・エル・シー Ｚｐ１５遺伝子座内への標的組込み
JP6215533B2 (ja) * 2009-04-09 2017-10-18 サンガモ セラピューティクス， インコーポレイテッド 幹細胞への標的組込み
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
EP3156504B1 (en) 2009-08-11 2018-10-31 Sangamo Therapeutics, 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
SG185367A1 (en) 2010-04-26 2012-12-28 Sangamo Biosciences Inc Genome editing of a rosa locus using zinc-finger nucleases
SI2566972T1 (sl) 2010-05-03 2020-04-30 Sangamo Therapeutics, Inc. Sestavki za povezovanje modulov cinkovega prsta
KR101953237B1 (ko) 2010-05-17 2019-02-28 상가모 테라퓨틱스, 인코포레이티드 신규 dna 결합 단백질 및 이의 용도
EP2596011B1 (en) 2010-07-21 2018-10-03 Sangamo Therapeutics, Inc. Methods and compositions for modification of a hla locus
EP2622090B1 (en) 2010-09-27 2019-06-19 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
EP2737063B1 (en) 2011-07-25 2016-06-01 Sangamo BioSciences, Inc. Methods and compositions for alteration of a cystic fibrosis transmembrane conductance regulator (cftr) gene
CA3186126A1 (en) 2011-09-21 2013-03-28 Sangamo Biosciences, Inc. Methods and compositions for regulation of transgene expression
CA2852955C (en) 2011-10-27 2021-02-16 Sangamo Biosciences, Inc. Methods and compositions for modification of the hprt locus
DK2839013T3 (da) * 2012-04-18 2020-09-14 Univ Leland Stanford Junior Ikke-disruptiv-gen-targetering
US9834786B2 (en) 2012-04-25 2017-12-05 Regeneron Pharmaceuticals, Inc. Nuclease-mediated targeting with large targeting vectors
CA2871524C (en) 2012-05-07 2021-07-27 Sangamo Biosciences, Inc. Methods and compositions for nuclease-mediated targeted integration of transgenes
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
US10648001B2 (en) 2012-07-11 2020-05-12 Sangamo Therapeutics, Inc. Method of treating mucopolysaccharidosis type I or II
WO2014011237A1 (en) 2012-07-11 2014-01-16 Sangamo Biosciences, Inc. Methods and compositions for the treatment of lysosomal storage diseases
CN104704110B (zh) 2012-08-29 2018-06-01 桑格摩生物科学股份有限公司 用于治疗遗传性病状的方法和组合物
JP6401704B2 (ja) 2012-10-10 2018-10-10 サンガモ セラピューティクス， インコーポレイテッド Ｔ細胞を修飾する化合物およびその使用
US20140140969A1 (en) * 2012-11-20 2014-05-22 Sangamo Biosciences, Inc. Methods and compositions for muscular dystrophies
HK1215873A1 (zh) 2012-12-05 2016-09-23 桑格摩治疗股份有限公司 調整代謝紊亂的方法及組合物
AU2013359091A1 (en) 2012-12-13 2015-07-02 Dow Agrosciences Llc Precision gene targeting to a particular locus in maize
US10227610B2 (en) 2013-02-25 2019-03-12 Sangamo Therapeutics, Inc. Methods and compositions for enhancing nuclease-mediated gene disruption
EP2975942B1 (en) 2013-03-21 2018-08-08 Sangamo Therapeutics, Inc. Targeted disruption of t cell receptor genes using engineered zinc finger protein nucleases
LT2986729T (lt) 2013-04-16 2018-10-25 Regeneron Pharmaceuticals, Inc. Žiurkės genomo tikslinė modifikacija
US10604771B2 (en) * 2013-05-10 2020-03-31 Sangamo Therapeutics, Inc. Delivery methods and compositions for nuclease-mediated genome engineering
JP2016521975A (ja) 2013-05-15 2016-07-28 サンガモ バイオサイエンシーズ， インコーポレイテッド 遺伝的状態の処置のための方法および組成物
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
AU2014337248B2 (en) 2013-10-17 2020-09-24 Sangamo Therapeutics, Inc. Delivery methods and compositions for nuclease-mediated genome engineering
US10117899B2 (en) 2013-10-17 2018-11-06 Sangamo Therapeutics, Inc. Delivery methods and compositions for nuclease-mediated genome engineering in hematopoietic stem cells
EP3068905A4 (en) 2013-11-11 2017-07-05 Sangamo BioSciences, Inc. Methods and compositions for treating huntington's disease
PT3068881T (pt) 2013-11-13 2019-05-31 Childrens Medical Center Regulação da expressão de genes mediada por nucleases
CN111330021A (zh) 2013-12-09 2020-06-26 桑格摩生物科学股份有限公司 基因组工程化的方法和组合物
CN110951779B (zh) 2013-12-11 2024-04-16 瑞泽恩制药公司 用于靶向修饰基因组的方法和组合物
US10072066B2 (en) 2014-02-03 2018-09-11 Sangamo Therapeutics, Inc. Methods and compositions for treatment of a beta thalessemia
KR20150096064A (ko) * 2014-02-14 2015-08-24 서울대학교산학협력단 Ｍｄｃｋ ⅱ 세포의 크로모좀에서 ａａｖｓ1 위치를 인식하고 이곳에 외래 유전자를 도입하여 안정하게 발현시키는 방법
JP6606088B2 (ja) * 2014-02-24 2019-11-13 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標的化組み込みのための方法および組成物
CA2942762C (en) 2014-03-18 2023-10-17 Sangamo Biosciences, Inc. Methods and compositions for regulation of zinc finger protein expression
ES2971516T3 (es) 2014-03-21 2024-06-05 Univ Leland Stanford Junior Edición del genoma sin nucleasas
WO2015164748A1 (en) 2014-04-24 2015-10-29 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
CN106413760B (zh) 2014-05-08 2020-01-14 桑格摩生物科学股份有限公司 用于治疗亨廷顿病的方法和组合物
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
SG11201609634YA (en) 2014-06-06 2016-12-29 Regeneron Pharma Methods and compositions for modifying a targeted locus
PL3161128T3 (pl) 2014-06-26 2019-02-28 Regeneron Pharmaceuticals, Inc. Sposoby i kompozycje do celowanych modyfikacji genetycznych i sposoby zastosowania
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
US9616090B2 (en) 2014-07-30 2017-04-11 Sangamo Biosciences, Inc. Gene correction of SCID-related genes in hematopoietic stem and progenitor cells
US10435677B2 (en) 2014-09-16 2019-10-08 Sangamo Therapeutics, Inc. Genetically modified human cell with a corrected mutant sickle cell mutation
DK3207124T3 (da) 2014-10-15 2019-08-12 Regeneron Pharma Fremgangsmåder og sammensætninger til generering eller bevaring af pluripotente celler
CA3176380A1 (en) 2014-11-21 2016-05-26 Regeneron Pharmaceuticals, Inc. Methods and compositions 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
EP3653048B9 (en) 2014-12-19 2023-10-04 Regeneron Pharmaceuticals, Inc. Methods and compositions for targeted genetic modification through single-step multiple targeting
HK1246690A1 (zh) 2015-01-21 2018-09-14 Sangamo Therapeutics, Inc. 用於鉴定高度特异性核酸酶的方法和组合物
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
US10179918B2 (en) 2015-05-07 2019-01-15 Sangamo Therapeutics, Inc. Methods and compositions for increasing transgene activity
US10808020B2 (en) * 2015-05-12 2020-10-20 Sangamo Therapeutics, Inc. Nuclease-mediated regulation of gene expression
US9957501B2 (en) 2015-06-18 2018-05-01 Sangamo Therapeutics, Inc. Nuclease-mediated regulation of gene expression
WO2017011519A1 (en) 2015-07-13 2017-01-19 Sangamo Biosciences, 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
CN108348576B (zh) 2015-09-23 2022-01-11 桑格摩生物治疗股份有限公司 Htt阻抑物及其用途
KR20250022906A (ko) 2015-10-28 2025-02-17 상가모 테라퓨틱스, 인코포레이티드 간-특이적 작제물인, 인자 viii 발현 카세트 및 이의 사용 방법
CA3004349A1 (en) 2015-11-23 2017-06-01 Sangamo Therapeutics, Inc. Methods and compositions for engineering immunity
WO2017106537A2 (en) 2015-12-18 2017-06-22 Sangamo Biosciences, Inc. Targeted disruption of the mhc cell receptor
JP7128741B2 (ja) 2015-12-18 2022-08-31 サンガモ セラピューティクス， インコーポレイテッド Ｔ細胞受容体の標的化破壊
WO2017123757A1 (en) 2016-01-15 2017-07-20 Sangamo Therapeutics, Inc. Methods and compositions for the treatment of neurologic disease
US10724020B2 (en) 2016-02-02 2020-07-28 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
ES2901989T3 (es) 2016-08-24 2022-03-24 Sangamo Therapeutics Inc Nucleasas específicas de objetivo modificadas
MX2019002207A (es) 2016-08-24 2019-07-15 Sangamo Therapeutics Inc Regulacion de la expresion genica usando nucleasas modificadas.
AU2017324462B2 (en) 2016-09-07 2024-03-21 Sangamo Therapeutics, Inc. Modulation of liver genes
CA3039673A1 (en) 2016-10-20 2018-04-26 Sangamo Therapeutics, Inc. Methods and compositions for the treatment of fabry disease
CA3041668A1 (en) 2016-10-31 2018-05-03 Sangamo Therapeutics, Inc. Gene correction of scid-related genes in hematopoietic stem and progenitor cells
CN110546265A (zh) * 2017-02-09 2019-12-06 因达普塔治疗公司 工程化自然杀伤(nk)细胞及其组合物和方法
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
EP3615510B1 (en) 2017-04-28 2024-03-27 Acuitas Therapeutics, Inc. Novel carbonyl lipids and lipid nanoparticle formulations for delivery of nucleic acids
US11655275B2 (en) 2017-05-03 2023-05-23 Sangamo Therapeutics, Inc. Methods and compositions for modification of a cystic fibrosis transmembrane conductance regulator (CFTR) gene
US11512287B2 (en) 2017-06-16 2022-11-29 Sangamo Therapeutics, Inc. Targeted disruption of T cell and/or HLA receptors
BR112019027673A2 (pt) 2017-07-31 2020-09-15 Regeneron Pharmaceuticals, Inc. animal não humano, e, métodos para testar a recombinação induzida por crispr/cas e para otimizar a capacidade de crispr/cas
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
CN115074343A (zh) 2017-07-31 2022-09-20 瑞泽恩制药公司 Cas转基因小鼠胚胎干细胞和小鼠及其应用
BR112020002438A2 (pt) 2017-08-08 2020-07-28 Sangamo Therapeutics, Inc. direcionamento celular mediado por receptores de antígenos quiméricos
IL274323B2 (en) 2017-11-09 2024-10-01 Sangamo Therapeutics Inc Genetic modification of cytokine inducible sh2-containing protein (cish) gene
CN111954540A (zh) 2018-02-08 2020-11-17 桑格摩生物治疗股份有限公司 工程化靶标特异性核酸酶
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
EP3592140A1 (en) 2018-03-19 2020-01-15 Regeneron Pharmaceuticals, Inc. Transcription modulation in animals using crispr/cas systems
MX2020010459A (es) 2018-04-05 2021-01-20 Juno Therapeutics Inc Metodos para producir celulas que expresan un receptor recombinante y composiciones relacionadas.
KR20210020873A (ko) 2018-04-05 2021-02-24 주노 쎄러퓨티크스 인코퍼레이티드 재조합 수용체를 발현하는 τ 세포, 관련 폴리뉴클레오티드 및 방법
EP3781587A4 (en) 2018-04-18 2022-03-30 Sangamo Therapeutics, Inc. ZINC FINGER PROTEIN COMPOSITIONS FOR THE MODULATION OF HUNTINGTINE (HTT)
US11690921B2 (en) 2018-05-18 2023-07-04 Sangamo Therapeutics, Inc. Delivery of target specific nucleases
WO2020041249A1 (en) 2018-08-23 2020-02-27 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
EP3853244A4 (en) 2018-09-18 2022-09-14 Sangamo Therapeutics, Inc. PROGRAMMED CELL DEATH 1 (PD1) SPECIFIC NUCLEASES
WO2020061426A2 (en) 2018-09-21 2020-03-26 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
CN113272428A (zh) 2018-10-18 2021-08-17 英特利亚治疗股份有限公司 核酸构建体和使用方法
WO2020082046A2 (en) 2018-10-18 2020-04-23 Intellia Therapeutics, Inc. Compositions and methods for expressing factor ix
CN113891934A (zh) 2018-11-21 2022-01-04 因达普塔治疗公司 扩增天然杀伤（nk）细胞子集的方法以及相关组合物和方法
CA3120570A1 (en) 2018-11-28 2020-06-04 Forty Seven, Inc. Genetically modified hspcs resistant to ablation regime
DK3908568T3 (da) 2019-01-11 2024-08-26 Acuitas Therapeutics Inc Lipider til lipidnanopartikellevering af aktivstoffer
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
CN118064502A (zh) 2019-04-03 2024-05-24 瑞泽恩制药公司 用于将抗体编码序列插入到安全港基因座中的方法和组合物
BR112021021200A2 (pt) 2019-05-01 2021-12-21 Juno Therapeutics Inc Células expressando um receptor quimérico de um locus cd247 modificado, polinucleotídeos relacionados e métodos
BR112021021075A2 (pt) 2019-05-01 2021-12-14 Editas Medicine Inc Células que expressam um receptor recombinante de um locus de tgfbr2 modificado, polinucleotídeos relacionados e métodos
BR112021026832A2 (pt) 2019-07-02 2022-05-10 Hutchinson Fred Cancer Res Vetores ad35 recombinantes e aprimoramentos da terapia gênica relacionada
US20230416776A1 (en) 2019-10-08 2023-12-28 Regents Of The University Of Minnesota Crispr-mediated human genome editing with vectors
EP4054651A1 (en) 2019-11-08 2022-09-14 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
US20230181641A1 (en) 2020-05-13 2023-06-15 Juno Therapeutics, Inc. Process for producing donor-batched cells expressing a recombinant receptor
EP4153739A1 (en) 2020-05-21 2023-03-29 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
CN115698335A (zh) 2020-05-22 2023-02-03 因斯特罗公司 使用机器学习模型预测疾病结果
KR20230042283A (ko) 2020-06-26 2023-03-28 주노 테라퓨틱스 게엠베하 재조합 수용체를 조건부로 발현하는 조작된 ｔ 세포, 관련된 폴리뉴클레오티드 및 방법
WO2022011099A1 (en) 2020-07-08 2022-01-13 Regents Of The University Of Minnesota Modified hexosaminidase and uses thereof
US11976019B2 (en) 2020-07-16 2024-05-07 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
CA3213080A1 (en) 2021-03-23 2022-09-29 Krit RITTHIPICHAI Cish gene editing of tumor infiltrating lymphocytes and uses of same in immunotherapy
WO2023035011A1 (en) 2021-09-03 2023-03-09 North Carolina State University Compositions and methods for conferring resistance to geminivirus
EP4423119A1 (en) 2021-10-27 2024-09-04 Regeneron Pharmaceuticals, Inc. Compositions and methods for expressing factor ix for hemophilia b therapy
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
AU2022408167A1 (en) 2021-12-08 2024-06-06 Regeneron Pharmaceuticals, Inc. Mutant myocilin disease model and uses thereof
KR20240123832A (ko) 2021-12-16 2024-08-14 아퀴타스 테라퓨틱스 인크. 지질 나노입자 제형에 사용하기 위한 지질
US20250032642A1 (en) 2022-02-02 2025-01-30 Regeneron Pharmaceuticals, Inc. Crispr-mediated transgene insertion in neonatal cells
EP4475671A1 (en) 2022-02-07 2024-12-18 Regeneron Pharmaceuticals, Inc. Compositions and methods for defining optimal treatment timeframes in lysosomal disease
US20250135039A1 (en) 2022-02-07 2025-05-01 Ensoma, Inc. Inhibitor-resistant mgmt modifications and modification of mgmt-encoding nucleic acids
US20250295810A1 (en) 2022-05-09 2025-09-25 Synteny Therapeutics, Inc. Erythroparvovirus with a modified capsid for gene therapy
US20250302994A1 (en) 2022-05-09 2025-10-02 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
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
IL319122A (en) 2022-09-28 2025-04-01 Regeneron Pharma Different antibody-resistant receptors improve cell-based therapies
IL320016A (en) 2022-11-04 2025-06-01 Regeneron Pharma 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
US20240173426A1 (en) 2022-11-14 2024-05-30 Regeneron Pharmaceuticals, Inc. Compositions and methods for fibroblast growth factor receptor 3-mediated delivery to astrocytes
AU2024214593A1 (en) 2023-02-03 2025-08-07 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
US12383615B2 (en) 2023-03-23 2025-08-12 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
WO2025029657A2 (en) 2023-07-28 2025-02-06 Regeneron Pharmaceuticals, Inc. Anti-tfr:gaa and anti-cd63:gaa insertion for treatment of pompe disease
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
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
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
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
WO2025265017A1 (en) 2024-06-20 2025-12-26 Regeneron Pharmaceuticals, Inc. Ass1 gene insertion for the treatment of citrullinemia type i

Family Cites Families (37)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5356802A (en) 1992-04-03 1994-10-18 The Johns Hopkins University Functional domains in flavobacterium okeanokoites (FokI) restriction endonuclease
US5436150A (en) 1992-04-03 1995-07-25 The Johns Hopkins University Functional domains in flavobacterium okeanokoities (foki) restriction endonuclease
US5487994A (en) 1992-04-03 1996-01-30 The Johns Hopkins University Insertion and deletion mutants of FokI restriction endonuclease
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
CA2181548C (en) 1994-01-18 2009-11-03 Carlos F. Barbas, Iii Zinc finger protein derivatives and methods therefor
AU698152B2 (en) 1994-08-20 1998-10-22 Gendaq Limited Improvements in or relating to 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
GB9703369D0 (en) 1997-02-18 1997-04-09 Lindqvist Bjorn H Process
GB2338237B (en) 1997-02-18 2001-02-28 Actinova Ltd In vitro peptide or protein expression library
GB9710809D0 (en) 1997-05-23 1997-07-23 Medical Res Council Nucleic acid binding proteins
GB9710807D0 (en) 1997-05-23 1997-07-23 Medical Res Council Nucleic acid binding proteins
US6410248B1 (en) 1998-01-30 2002-06-25 Massachusetts Institute Of Technology General strategy for selecting high-affinity zinc finger proteins for diverse DNA target sites
DE69942334D1 (de) 1998-03-02 2010-06-17 Massachusetts Inst Technology Poly-zinkfinger-proteine mit verbesserten linkern
US6140081A (en) 1998-10-16 2000-10-31 The Scripps Research Institute Zinc finger binding domains for GNN
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
AU2001263155A1 (en) 2000-05-16 2001-11-26 Massachusetts Institute Of Technology Methods and compositions for interaction trap assays
JP2002060786A (ja) 2000-08-23 2002-02-26 Kao Corp 硬質表面用殺菌防汚剤
AU2002241946B2 (en) 2001-01-22 2007-04-26 Sangamo Therapeutics, Inc. Modified zinc finger binding proteins
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
CN100575485C (zh) 2002-01-23 2009-12-30 犹他大学研究基金会 使用锌指核酸酶的定向染色体诱变
WO2003080809A2 (en) 2002-03-21 2003-10-02 Sangamo Biosciences, Inc. Methods and compositions for using zinc finger endonucleases to enhance homologous recombination
CA2497913C (en) 2002-09-05 2014-06-03 California Institute Of Technology Use of chimeric nucleases to stimulate gene targeting
US8409861B2 (en) 2003-08-08 2013-04-02 Sangamo Biosciences, Inc. Targeted deletion of cellular DNA sequences
EP3222715A1 (en) * 2003-08-08 2017-09-27 Sangamo BioSciences, Inc. Methods and compositions for targeted cleavage and recombination
US7888121B2 (en) 2003-08-08 2011-02-15 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
AU2005287278B2 (en) 2004-09-16 2011-08-04 Sangamo Biosciences, Inc. Compositions and methods for protein production
WO2006121579A2 (en) 2005-04-18 2006-11-16 Mount Sinai School Of Medicine Of New York University Targeted gene addition in stem cells
CN101273141B (zh) * 2005-07-26 2013-03-27 桑格摩生物科学股份有限公司 外源核酸序列的靶向整合和表达
AU2007267887B2 (en) 2006-05-25 2013-05-30 Sangamo Therapeutics, Inc. Variant Foki Cleavage Half-Domains

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