ATE489465T1 - Gezielte integration in die ppp1r12c-position - Google Patents

Gezielte integration in die ppp1r12c-position

Info

Publication number
ATE489465T1
ATE489465T1 AT08743239T AT08743239T ATE489465T1 AT E489465 T1 ATE489465 T1 AT E489465T1 AT 08743239 T AT08743239 T AT 08743239T AT 08743239 T AT08743239 T AT 08743239T AT E489465 T1 ATE489465 T1 AT E489465T1
Authority
AT
Austria
Prior art keywords
targeted integration
ppp1r12c
polypeptide
interest
expression
Prior art date
Application number
AT08743239T
Other languages
German (de)
English (en)
Inventor
Russell Dekelver
Philip D Gregory
David Paschon
Fyodor Urnov
Phillip Tam
Original Assignee
Sangamo Biosciences Inc
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Application filed by Sangamo Biosciences Inc filed Critical Sangamo Biosciences Inc
Application granted granted Critical
Publication of ATE489465T1 publication Critical patent/ATE489465T1/de

Links

Classifications

    • 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
    • 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
    • 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
    • C12N15/11—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

Landscapes

  • 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)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Toxicology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Virology (AREA)
  • Bioinformatics & Computational Biology (AREA)
  • Mycology (AREA)
  • Cell Biology (AREA)
  • Tropical Medicine & Parasitology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (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)
AT08743239T 2007-04-26 2008-04-24 Gezielte integration in die ppp1r12c-position ATE489465T1 (de)

Applications Claiming Priority (2)

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

Publications (1)

Publication Number Publication Date
ATE489465T1 true ATE489465T1 (de) 2010-12-15

Family

ID=39926269

Family Applications (1)

Application Number Title Priority Date Filing Date
AT08743239T ATE489465T1 (de) 2007-04-26 2008-04-24 Gezielte integration in die ppp1r12c-position

Country Status (9)

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

Families Citing this family (166)

* 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
WO2010053518A2 (en) * 2008-10-29 2010-05-14 Sangamo Biosciences, Inc. Methods and compositions for inactivating glutamine synthetase gene expression
EP3156494B8 (en) 2008-12-04 2018-09-19 Sangamo Therapeutics, Inc. Genome editing in rats using zinc-finger nucleases
KR101683928B1 (ko) * 2008-12-17 2016-12-07 다우 아그로사이언시즈 엘엘씨 Ｚｐ１５ 유전자 자리 내로의 표적화 통합
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
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
KR102262704B1 (ko) 2009-08-11 2021-06-09 상가모 테라퓨틱스, 인코포레이티드 표적화 변형에 대한 동형접합성 유기체
US8956828B2 (en) 2009-11-10 2015-02-17 Sangamo Biosciences, Inc. Targeted disruption of T cell receptor genes using engineered zinc finger protein nucleases
KR101866578B1 (ko) * 2010-01-22 2018-06-11 다우 아그로사이언시즈 엘엘씨 표적화된 게놈 교체
EP2534163B1 (en) 2010-02-09 2015-11-04 Sangamo BioSciences, Inc. Targeted genomic modification with partially single-stranded donor molecules
US8771985B2 (en) 2010-04-26 2014-07-08 Sangamo Biosciences, Inc. Genome editing of a Rosa locus using zinc-finger nucleases
PT2566972T (pt) 2010-05-03 2020-03-02 Sangamo Biosciences Inc Composições para a ligação de módulos de dedo de zinco
JP6208580B2 (ja) 2010-05-17 2017-10-04 サンガモ セラピューティクス， インコーポレイテッド 新規のｄｎａ結合タンパク質及びその使用
CA2993567C (en) 2010-07-21 2022-06-28 Sangamo Biosciences, Inc. Methods and compositions for modification of a t-cell receptor gene
CA2811364C (en) 2010-09-27 2022-01-04 Sangamo Biosciences, Inc. Methods and compositions for inhibiting viral entry into cells
EP2627665B1 (en) 2010-10-12 2015-12-16 The Children's Hospital Of Philadelphia Methods and compositions for treating hemophilia B
US9161995B2 (en) 2011-07-25 2015-10-20 Sangamo Biosciences, Inc. Methods and compositions for alteration of a cystic fibrosis transmembrane conductance regulator (CFTR) gene
AU2012312260B2 (en) 2011-09-21 2017-08-31 Sangamo Therapeutics, Inc. Methods and compositions for regulation of transgene expression
JP6188703B2 (ja) 2011-10-27 2017-08-30 サンガモ セラピューティクス， インコーポレイテッド Ｈｐｒｔ遺伝子座を修飾するための方法および組成物
WO2013158309A2 (en) * 2012-04-18 2013-10-24 The Board Of Trustees Of The Leland Stanford Junior University Non-disruptive gene targeting
IN2014DN09261A (https=) 2012-04-25 2015-07-10 Regeneron Pharma
AU2013259647B2 (en) 2012-05-07 2018-11-08 Corteva Agriscience Llc Methods and compositions for nuclease-mediated targeted integration of transgenes
DK3444342T3 (da) 2012-07-11 2020-08-24 Sangamo Therapeutics Inc Fremgangsmåder og sammensætninger til behandlingen af lysosomale aflejringssygdomme
US10648001B2 (en) 2012-07-11 2020-05-12 Sangamo Therapeutics, Inc. Method of treating mucopolysaccharidosis type I or II
WO2014011901A2 (en) 2012-07-11 2014-01-16 Sangamo Biosciences, Inc. Methods and compositions for delivery of biologics
SI2890780T1 (sl) 2012-08-29 2020-11-30 Sangamo Therapeutics, Inc. Postopki in sestavki za zdravljenje genetskega stanja
EP2906684B8 (en) 2012-10-10 2020-09-02 Sangamo Therapeutics, 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
WO2014089212A1 (en) * 2012-12-05 2014-06-12 Sangamo Biosciences, Inc. Methods and compositions for regulation of metabolic disorders
CA2895117A1 (en) 2012-12-13 2014-06-19 James W. Bing 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
JP6346266B2 (ja) 2013-03-21 2018-06-20 サンガモ セラピューティクス， インコーポレイテッド 操作されたジンクフィンガータンパク質ヌクレアーゼを使用するｔ細胞受容体遺伝子の標的化された破壊
SI2986729T1 (sl) 2013-04-16 2019-02-28 Regeneron Pharmaceuticals, Inc. Ciljana sprememba genoma podgane
US10604771B2 (en) * 2013-05-10 2020-03-31 Sangamo Therapeutics, Inc. Delivery methods and compositions for nuclease-mediated genome engineering
CN105683376A (zh) 2013-05-15 2016-06-15 桑格摩生物科学股份有限公司 用于治疗遗传病状的方法和组合物
AU2014312295C1 (en) 2013-08-28 2020-08-13 Sangamo Therapeutics, Inc. Compositions for linking DNA-binding domains and cleavage domains
CN105899665B (zh) * 2013-10-17 2019-10-22 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
US10117899B2 (en) 2013-10-17 2018-11-06 Sangamo Therapeutics, Inc. Delivery methods and compositions for nuclease-mediated genome engineering in hematopoietic stem cells
WO2015070212A1 (en) 2013-11-11 2015-05-14 Sangamo Biosciences, Inc. Methods and compositions for treating huntington's disease
HUE044540T2 (hu) 2013-11-13 2019-10-28 Childrens Medical Center Nukleáz közvetítette génexpresszió-szabályozás
DK3080274T3 (da) 2013-12-09 2020-08-31 Sangamo Therapeutics Inc Fremgangsmåder og sammensætninger til genom-manipulation
US9546384B2 (en) 2013-12-11 2017-01-17 Regeneron Pharmaceuticals, Inc. Methods and compositions for the targeted modification of a mouse genome
WO2015117081A2 (en) 2014-02-03 2015-08-06 Sangamo Biosciences, 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
AU2015218576B2 (en) * 2014-02-24 2020-02-27 Sangamo Therapeutics, Inc. Methods and compositions for nuclease-mediated targeted integration
CN106459894B (zh) 2014-03-18 2020-02-18 桑格摩生物科学股份有限公司 用于调控锌指蛋白表达的方法和组合物
BR112016019940A2 (pt) 2014-03-21 2017-10-24 Univ Leland Stanford Junior edição de genoma sem nucleases
WO2015164748A1 (en) 2014-04-24 2015-10-29 Sangamo Biosciences, Inc. Engineered transcription activator like effector (tale) proteins
CN107405411A (zh) * 2014-05-01 2017-11-28 华盛顿大学 使用腺病毒载体的体内基因改造
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
RU2691102C2 (ru) 2014-05-08 2019-06-11 Сангамо Байосайенсиз, Инк. Способы и композиции для лечения болезни хантингтона
AU2015259191B2 (en) 2014-05-13 2019-03-21 Sangamo Therapeutics, 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
RS60359B1 (sr) 2014-06-06 2020-07-31 Regeneron Pharma Postupci i kompozicije za modifikovanje ciljanog lokusa
KR102386101B1 (ko) 2014-06-26 2022-04-14 리제너론 파마슈티칼스 인코포레이티드 표적화된 유전자 변형을 위한 방법 및 그 조성물, 및 사용 방법
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
HUE055583T2 (hu) 2014-09-16 2021-12-28 Sangamo Therapeutics Inc Eljárások és készítmények nukleáz által közvetített genommódosításhoz és -javításhoz hematopoetikus õssejtekben
DK3207124T3 (da) 2014-10-15 2019-08-12 Regeneron Pharma Fremgangsmåder og sammensætninger til generering eller bevaring af pluripotente celler
ES2731437T3 (es) 2014-11-21 2019-11-15 Regeneron Pharma Métodos y composiciones para la modificación genética dirigida mediante el uso de pares de ARN guías
US10889834B2 (en) 2014-12-15 2021-01-12 Sangamo Therapeutics, Inc. Methods and compositions for enhancing targeted transgene integration
KR102530821B1 (ko) 2014-12-19 2023-05-10 리제너론 파마슈티칼스 인코포레이티드 단일 단계 다중 표적화를 통한 표적화된 유전자 변형을 위한 방법 및 조성물
EP3247366A4 (en) 2015-01-21 2018-10-31 Sangamo Therapeutics, Inc. Methods and compositions for identification of highly specific nucleases
EP3256578B8 (en) 2015-02-13 2020-04-01 INSERM - Institut National de la Santé et de la Recherche Médicale 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
EP3322297B1 (en) 2015-07-13 2024-12-04 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
WO2017053753A1 (en) 2015-09-23 2017-03-30 Sangamo Biosciences, Inc. Htt repressors and uses thereof
CA3002524A1 (en) 2015-10-28 2017-05-04 Sangamo Therapeutics, Inc. Liver-specific constructs, factor viii expression cassettes and methods of use thereof
US10639383B2 (en) 2015-11-23 2020-05-05 Sangamo Therapeutics, Inc. Methods and compositions for engineering immunity
IL297018A (en) 2015-12-18 2022-12-01 Sangamo Therapeutics Inc Directed cleavage of cell mhc receptor
AU2016369490C1 (en) 2015-12-18 2021-12-23 Sangamo Therapeutics, Inc. Targeted disruption of the T cell receptor
CA3010738A1 (en) 2016-01-15 2017-07-20 Sangamo Therapeutics, Inc. Methods and compositions for the treatment of neurologic disease
EP3411056A4 (en) 2016-02-02 2019-10-02 Sangamo Therapeutics, Inc. COMPOUNDS FOR NETWORKING DNA BINDING DOMAINS AND SPLITTING 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
LT3504229T (lt) 2016-08-24 2021-12-10 Sangamo Therapeutics, Inc. Genų raiškos reguliavimas, panaudojant rekombinantines nukleazes
SG10201913948PA (en) 2016-08-24 2020-03-30 Sangamo Therapeutics Inc Engineered target specific nucleases
WO2018049009A2 (en) 2016-09-07 2018-03-15 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
JP7108608B2 (ja) 2016-10-31 2022-07-28 サンガモ セラピューティクス， インコーポレイテッド 造血幹細胞および前駆細胞におけるｓｃｉｄ関連遺伝子の遺伝子修正
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
WO2018200943A1 (en) 2017-04-28 2018-11-01 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
CA3068634A1 (en) 2017-06-30 2019-01-03 Memorial Sloan Kettering Cancer Center Compositions and methods for adoptive cell therapy for cancer
WO2019028032A1 (en) 2017-07-31 2019-02-07 Regeneron Pharmaceuticals, Inc. EMBRYONIC STEM CELLS OF TRANSGENIC MOUSE CASES AND MICE AND USES THEREOF
CN111182790A (zh) 2017-07-31 2020-05-19 瑞泽恩制药公司 Crispr报告体非人类动物及其用途
CN110891419A (zh) 2017-07-31 2020-03-17 瑞泽恩制药公司 评价crispr/cas-诱导的与外源供体核酸的体内重组
CA3071683A1 (en) 2017-08-08 2019-02-14 Sangamo Therapeutics, Inc. Chimeric antigen receptor mediated cell targeting
EP3706766A4 (en) 2017-11-09 2021-08-18 Sangamo Therapeutics, Inc. GENETIC MODIFICATION OF THE CYTOCINE INDUCTIBLE SH2-CONTAINING PROTEIN (CISH) GENE
CA3089587A1 (en) 2018-02-08 2019-08-15 Sangamo Therapeutics, Inc. Engineered target specific nucleases
CA3091143A1 (en) 2018-02-15 2019-08-22 Memorial Sloan-Kettering Cancer Center Foxp3 targeting agent compositions and methods of use for adoptive cell therapy
CN111885915B (zh) 2018-03-19 2023-04-28 瑞泽恩制药公司 使用crispr/cas系统对动物进行转录调制
KR20210020873A (ko) 2018-04-05 2021-02-24 주노 쎄러퓨티크스 인코퍼레이티드 재조합 수용체를 발현하는 τ 세포, 관련 폴리뉴클레오티드 및 방법
CA3094468A1 (en) 2018-04-05 2019-10-10 Juno Therapeutics, Inc. Methods of producing cells expressing a recombinant receptor and related compositions
WO2019204457A1 (en) 2018-04-18 2019-10-24 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
JP7541508B2 (ja) 2018-08-23 2024-08-28 サンガモ セラピューティクス， インコーポレイテッド 操作された標的特異的な塩基エディター
EP3849565A4 (en) 2018-09-12 2022-12-28 Fred Hutchinson Cancer Research Center REDUCTION OF CD33 EXPRESSION TO SELECTIVELY PROTECT THERAPEUTIC CELLS
WO2020061161A1 (en) 2018-09-18 2020-03-26 Sangamo Therapeutics, Inc. Programmed cell death 1 (pd1) specific nucleases
IL281615B2 (en) 2018-09-21 2026-01-01 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
JP7543259B2 (ja) 2018-10-18 2024-09-02 アクイタス セラピューティクス インコーポレイテッド 活性剤の脂質ナノ粒子送達のための脂質
US20200268906A1 (en) 2018-10-18 2020-08-27 Intellia Therapeutics, Inc. Nucleic acid constructs and methods of use
JP7578590B2 (ja) 2018-10-18 2024-11-06 インテリア セラピューティクス，インコーポレーテッド 第ｉｘ因子を発現するための組成物及び方法
US12410402B2 (en) 2018-11-21 2025-09-09 Indapta Therapeutics, Inc. Methods for expansion of natural killer (NK) cell subset and related compositions and methods
US20220023348A1 (en) 2018-11-28 2022-01-27 Forty Seven, Inc. Genetically modified hspcs resistant to ablation regime
MY202837A (en) 2019-01-11 2024-05-24 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
US20220154157A1 (en) 2019-02-06 2022-05-19 Emendobio Inc. New engineered high fidelity cas9
WO2020206162A1 (en) 2019-04-03 2020-10-08 Regeneron Pharmaceuticals, Inc. Methods and compositions for insertion of antibody coding sequences into a safe harbor locus
AU2020265741A1 (en) 2019-05-01 2021-11-25 Editas Medicine, Inc. Cells expressing a recombinant receptor from a modified TGFBR2 Locus, related polynucleotides and methods
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
JP2022539248A (ja) 2019-07-02 2022-09-07 フレッド ハッチンソン キャンサー リサーチ センター 組換えａｄ３５ベクター及び関連遺伝子治療改善
WO2021072115A1 (en) 2019-10-08 2021-04-15 Regents Of The University Of Minnesota Crispr-mediated human genome editing with vectors
US12521451B2 (en) 2019-11-08 2026-01-13 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
WO2021231661A2 (en) 2020-05-13 2021-11-18 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
GB202007578D0 (en) 2020-05-21 2020-07-08 Univ Oxford Innovation Ltd Hdr enhancers
EP4153741A1 (en) 2020-05-21 2023-03-29 Oxford Genetics Limited Hdr enhancers
AU2021275995B2 (en) 2020-05-22 2026-02-12 Insitro, Inc. Predicting disease outcomes using machine learned models
EP4171616A1 (en) 2020-06-26 2023-05-03 Juno Therapeutics GmbH Engineered t cells conditionally expressing a recombinant receptor, related polynucleotides and methods
US20230374483A1 (en) 2020-07-08 2023-11-23 Regents Of The University Of Minnesota Modified hexosaminidase and uses thereof
WO2022016070A1 (en) 2020-07-16 2022-01-20 Acuitas Therapeutics, Inc. Cationic lipids for use in lipid nanoparticles
JP2023549780A (ja) 2020-11-04 2023-11-29 ジュノー セラピューティクス インコーポレイテッド 改変されたインバリアントｃｄ３免疫グロブリンスーパーファミリー鎖遺伝子座からキメラ受容体を発現する細胞ならびに関連するポリヌクレオチドおよび方法
AR125199A1 (es) 2021-03-23 2023-06-21 Iovance Biotherapeutics Inc Edición génica cish de linfocitos infiltrantes de tumores y usos de los mismos en inmunoterapia
WO2023035011A1 (en) 2021-09-03 2023-03-09 North Carolina State University Compositions and methods for conferring resistance to geminivirus
US20230149563A1 (en) 2021-10-27 2023-05-18 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
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
CA3242402A1 (en) 2021-12-16 2023-06-22 Acuitas Therapeutics, Inc. Lipids for use in lipid nanoparticle formulations
EP4473103A2 (en) 2022-02-02 2024-12-11 Regeneron Pharmaceuticals, Inc. Anti-tfr:gaa and anti-cd63:gaa insertions for treatment of pompe disease
US20250194571A1 (en) 2022-02-07 2025-06-19 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
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
EP4522210A1 (en) 2022-05-09 2025-03-19 Synteny Therapeutics, Inc. Erythroparvovirus with a modified genome for gene therapy
IL318625A (en) 2022-07-29 2025-03-01 Regeneron Pharma Compositions and methods for transferrin receptor (TFR)-mediated delivery to brain and muscle
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
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
AU2023379457A1 (en) 2022-11-14 2025-05-15 Regeneron Pharmaceuticals, Inc. Compositions and methods for fibroblast growth factor receptor 3-mediated delivery to astrocytes
CN121002052A (zh) 2023-02-03 2025-11-21 C3S2 有限公司 用于非病毒生产工程化免疫细胞的方法
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
EP4683931A1 (en) 2023-03-23 2026-01-28 Carbon Biosciences, Inc. Parvovirus compositions and related methods for gene therapy
AU2024315073A1 (en) 2023-07-28 2026-01-22 Regeneron Pharmaceuticals, Inc. Use of bgh-sv40l tandem polya to enhance transgene expression during unidirectional gene insertion
AU2024317483A1 (en) 2023-07-28 2026-01-29 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
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
WO2026072529A1 (en) 2024-09-24 2026-04-02 University Of Florida Research Foundation, Incorporated Mettl7a for improved embryo competence
WO2026072721A1 (en) 2024-09-25 2026-04-02 Carbon Biosciences, Inc. Parvovirus compositions and methods for gene therapy
WO2026073244A1 (en) 2024-09-30 2026-04-02 Ensoma, Inc. Constructs for multi-lineage expression of therapeutic agents

Family Cites Families (37)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
US5356802A (en) * 1992-04-03 1994-10-18 The Johns Hopkins University Functional domains in flavobacterium okeanokoites (FokI) restriction endonuclease
US6140466A (en) * 1994-01-18 2000-10-31 The Scripps Research Institute Zinc finger protein derivatives and methods therefor
DE69534629D1 (de) 1994-01-18 2005-12-29 Scripps Research Inst Derivate von zinkfingerproteinen und methoden
US6242568B1 (en) * 1994-01-18 2001-06-05 The Scripps Research Institute Zinc finger protein derivatives and methods therefor
ATE524545T1 (de) 1994-08-20 2011-09-15 Gendaq Ltd Verbesserungen bei oder im zusammenhang mit bindeproteinen zur erkennung von dna
GB9824544D0 (en) 1998-11-09 1999-01-06 Medical Res Council Screening system
US5789538A (en) * 1995-02-03 1998-08-04 Massachusetts Institute Of Technology Zinc finger proteins with high affinity new DNA binding specificities
US5925523A (en) * 1996-08-23 1999-07-20 President & Fellows Of Harvard College Intraction trap assay, reagents and uses thereof
GB2338237B (en) 1997-02-18 2001-02-28 Actinova Ltd In vitro peptide or protein expression library
GB9703369D0 (en) 1997-02-18 1997-04-09 Lindqvist Bjorn H Process
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
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
WO2001053480A1 (en) 2000-01-24 2001-07-26 Sangamo Biosciences, Inc. 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
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
WO2002057294A2 (en) 2001-01-22 2002-07-25 Sangamo Biosciences, Inc. Zinc finger proteins for dna binding and gene regulation in plants
GB0108491D0 (en) 2001-04-04 2001-05-23 Gendaq Ltd Engineering zinc fingers
US20040224385A1 (en) 2001-08-20 2004-11-11 Barbas Carlos F Zinc finger binding domains for cnn
EP1476547B1 (en) * 2002-01-23 2006-12-06 The University of Utah Research Foundation Targeted chromosomal mutagenesis using zinc finger nucleases
US20030232410A1 (en) * 2002-03-21 2003-12-18 Monika Liljedahl Methods and compositions for using zinc finger endonucleases to enhance homologous recombination
EP2806025B1 (en) * 2002-09-05 2019-04-03 California Institute of Technology Use of zinc finger nucleases to stimulate gene targeting
US8409861B2 (en) * 2003-08-08 2013-04-02 Sangamo Biosciences, Inc. Targeted deletion of cellular DNA sequences
JP4555292B2 (ja) * 2003-08-08 2010-09-29 サンガモ バイオサイエンシズ インコーポレイテッド 標的化された切断及び組換えの方法及び組成物
US7888121B2 (en) * 2003-08-08 2011-02-15 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
KR20070060115A (ko) * 2004-09-16 2007-06-12 상가모 바이오사이언스 인코포레이티드 단백질 생산을 위한 조성물 및 방법
CA2605324A1 (en) 2005-04-18 2006-11-16 Mount Sinai School Of Medicine Of New York University Targeted gene addition in stem cells
AU2006272634B2 (en) 2005-07-26 2013-01-24 Sangamo Therapeutics, Inc. Targeted integration and expression of exogenous nucleic acid sequences
JP5266210B2 (ja) 2006-05-25 2013-08-21 サンガモ バイオサイエンシズ インコーポレイテッド 改変開裂ハーフドメイン

Also Published As

Publication number Publication date
DE602008003684D1 (de) 2011-01-05
WO2008133938A3 (en) 2009-04-02
HK1136847A1 (en) 2010-07-09
AU2008244473B2 (en) 2013-06-20
AU2008244473A1 (en) 2008-11-06
US8110379B2 (en) 2012-02-07
CA2684378C (en) 2016-11-29
JP5400034B2 (ja) 2014-01-29
US11649468B2 (en) 2023-05-16
US9267154B2 (en) 2016-02-23
CN101679977A (zh) 2010-03-24
US20160289699A1 (en) 2016-10-06
WO2008133938A2 (en) 2008-11-06
US8822221B2 (en) 2014-09-02
US20120142055A1 (en) 2012-06-07
US20180201955A1 (en) 2018-07-19
US20080299580A1 (en) 2008-12-04
EP2137310A2 (en) 2009-12-30
CN101679977B (zh) 2013-05-01
JP2010524500A (ja) 2010-07-22
EP2137310B1 (en) 2010-11-24
CA2684378A1 (en) 2008-11-06
US9914940B2 (en) 2018-03-13
US20140349394A1 (en) 2014-11-27

Similar Documents

Publication Publication Date Title
ATE489465T1 (de) Gezielte integration in die ppp1r12c-position
MX2010008632A (es) Leptina-polipeptidos modificados y sus usos.
LTC1971366I2 (lt) Žmogaus anti-IL-23 antikūnai, kompozicijos, metodai ir panaudojimai
CO6561839A2 (es) Alteracion genómica dirigida
TR201821029T4 (tr) Geliştirilmiş antı-IL-23p19 antikorları.
WO2010011735A3 (en) Modified bovine g-csf polypeptides and their uses
FR2883179B1 (fr) Comprime enrobe
UA104459C2 (uk) Антитіла проти fgfr3 і способи їх застосування
MY148635A (en) Anti-tat226 antibodies and immunoconjugates
CY1116261T1 (el) Αντισωματα και ανοσοσυζευγματα και χρησεις αυτων
MX2010006576A (es) Composiciones y metodos para producir isopreno.
EP4302783A3 (en) Modified relaxin polypeptides and their uses
EA201101520A1 (ru) Способы диагностики онкологических заболеваний, использующие эпиметаболические переключатели, многоаспектные внутриклеточные молекулы или факторы влияния
EA200802008A1 (ru) Фармацевтические композиции
DE112007000745A5 (de) Die Tornadormaschine - Ein Nebel-Wirbler, der als Effektgerät, Luftbefeuchter und Aerosolgenerator einsetzbar ist
EA201070447A1 (ru) Способ обработки волос
CA125780S (en) Air freshener device
BRPI0819649A2 (pt) método, organismo geneticamente modificado, e, célula de levedura recinbinante
MX2009004030A (es) Articulos antimicrobianos y metodos de fabricacion.
ATE526394T1 (de) Verfahren zur expansion von cd4+ cd25+ t regulatory zellen
WO2010056144A3 (en) Foxp3+ natural kiler t-cells and the treatment of immune related diseases
IN2012DN01538A (https=)
WO2010036344A3 (en) Compositions, oral care products and methods of making and using the same
BRPI0921371A2 (pt) Células-tronco para uso terapêutico que são derivados de monócito humano, e método para induzir as mesmas.
ATE512185T1 (de) Heterobifunktionelle polyethylenglycol-reagenzien

Legal Events

Date Code Title Description
RER Ceased as to paragraph 5 lit. 3 law introducing patent treaties