HK1136847A1 - Targeted integration into the ppp1r12c locus - Google Patents

Targeted integration into the ppp1r12c locus

Info

Publication number
HK1136847A1
HK1136847A1 HK10104464.7A HK10104464A HK1136847A1 HK 1136847 A1 HK1136847 A1 HK 1136847A1 HK 10104464 A HK10104464 A HK 10104464A HK 1136847 A1 HK1136847 A1 HK 1136847A1
Authority
HK
Hong Kong
Prior art keywords
targeted integration
ppp1r12c locus
locus
ppp1r12c
targeted
Prior art date
Application number
HK10104464.7A
Other languages
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
Publication of HK1136847A1 publication Critical patent/HK1136847A1/xx

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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
    • C12N15/87Introduction 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/43504Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from invertebrates
    • C07K14/43595Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from invertebrates from coelenteratae, e.g. medusae
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    • C12N15/09Recombinant DNA-technology
    • 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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    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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 RNAses, DNAses
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    • C12YENZYMES
    • 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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    • C07ORGANIC CHEMISTRY
    • 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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  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Virology (AREA)
  • Bioinformatics & Computational Biology (AREA)
  • Cell Biology (AREA)
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  • Tropical Medicine & Parasitology (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Public Health (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Oscillators With Electromechanical Resonators (AREA)
HK10104464.7A 2007-04-26 2010-05-07 Targeted integration into the ppp1r12c locus HK1136847A1 (en)

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
HK1136847A1 true HK1136847A1 (en) 2010-07-09

Family

ID=39926269

Family Applications (1)

Application Number Title Priority Date Filing Date
HK10104464.7A HK1136847A1 (en) 2007-04-26 2010-05-07 Targeted integration into the ppp1r12c locus

Country Status (10)

Country Link
US (5) US8110379B2 (xx)
EP (1) EP2137310B1 (xx)
JP (1) JP5400034B2 (xx)
CN (1) CN101679977B (xx)
AT (1) ATE489465T1 (xx)
AU (1) AU2008244473B2 (xx)
CA (1) CA2684378C (xx)
DE (1) DE602008003684D1 (xx)
HK (1) HK1136847A1 (xx)
WO (1) WO2008133938A2 (xx)

Families Citing this family (141)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2205752B1 (en) 2007-10-25 2016-08-10 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
AU2009311697B2 (en) * 2008-10-29 2014-12-18 Sangamo Therapeutics, Inc. Methods and compositions for inactivating glutamine synthetase gene expression
EP2352369B1 (en) 2008-12-04 2017-04-26 Sangamo BioSciences, Inc. Genome editing in rats using zinc-finger nucleases
PT2370575T (pt) 2008-12-17 2017-12-11 Dow Agrosciences Llc Integração dirigida no locus zp15
WO2010117464A1 (en) 2009-04-09 2010-10-14 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
JP5902631B2 (ja) * 2010-01-22 2016-04-13 ダウ アグロサイエンシィズ エルエルシー 標的化ゲノム変更
EP2534163B1 (en) 2010-02-09 2015-11-04 Sangamo BioSciences, Inc. Targeted genomic modification with partially single-stranded donor molecules
KR101880536B1 (ko) 2010-04-26 2018-07-23 상가모 테라퓨틱스, 인코포레이티드 아연 핑거 뉴클레아제를 사용하는 로사 좌위의 게놈 편집
SI2566972T1 (sl) 2010-05-03 2020-04-30 Sangamo Therapeutics, Inc. Sestavki za povezovanje modulov cinkovega prsta
EP2571512B1 (en) 2010-05-17 2017-08-23 Sangamo BioSciences, Inc. Novel dna-binding proteins and uses thereof
JP6050230B2 (ja) 2010-07-21 2016-12-21 サンガモ バイオサイエンシーズ, インコーポレイテッド Hla遺伝子座の修飾のための方法及び組成物
AU2011312562B2 (en) 2010-09-27 2014-10-09 Sangamo Therapeutics, Inc. Methods and compositions for inhibiting viral entry into cells
KR101930711B1 (ko) 2010-10-12 2018-12-19 더 칠드런스 호스피탈 오브 필라델피아 혈우병 b의 치료를 위한 방법 및 조성물
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
ES2961613T3 (es) 2011-09-21 2024-03-12 Sangamo Therapeutics Inc Métodos y composiciones para la regulación de la expresión transgénica
CA3099582A1 (en) 2011-10-27 2013-05-02 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
SG10201702445TA (en) 2012-04-25 2017-04-27 Regeneron Pharma Nuclease-mediated targeting with large targeting vectors
EP2847338B1 (en) 2012-05-07 2018-09-19 Sangamo Therapeutics, Inc. Methods and compositions for nuclease-mediated targeted integration of transgenes
WO2014011901A2 (en) 2012-07-11 2014-01-16 Sangamo Biosciences, Inc. Methods and compositions for delivery of biologics
US10648001B2 (en) 2012-07-11 2020-05-12 Sangamo Therapeutics, Inc. Method of treating mucopolysaccharidosis type I or II
ES2613691T3 (es) 2012-07-11 2017-05-25 Sangamo Biosciences, Inc. Métodos y composiciones para el tratamiento de enfermedades por almacenamiento lisosomal
AU2013308770B2 (en) 2012-08-29 2019-01-17 Sangamo Therapeutics, Inc. Methods and compositions for treatment of a genetic condition
ES2824024T3 (es) 2012-10-10 2021-05-11 Sangamo Therapeutics Inc Compuestos modificadores de células T y usos de los mismos
US20140140969A1 (en) * 2012-11-20 2014-05-22 Sangamo Biosciences, Inc. Methods and compositions for muscular dystrophies
JP2016500254A (ja) 2012-12-05 2016-01-12 サンガモ バイオサイエンシーズ, インコーポレイテッド 代謝疾患の調節のための方法および組成物
CA2895117A1 (en) 2012-12-13 2014-06-19 James W. Bing Precision gene targeting to a particular locus in maize
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
AU2014235968B2 (en) 2013-03-21 2018-05-24 Ospedale San Raffaele Srl Targeted disruption of T cell receptor genes using engineered zinc finger protein nucleases
HUE056903T2 (hu) 2013-04-16 2022-04-28 Regeneron Pharma Patkány genom célzott módosítása
CA2910427C (en) * 2013-05-10 2024-02-20 Sangamo Biosciences, Inc. Delivery methods and compositions for nuclease-mediated genome engineering
CN116083487A (zh) 2013-05-15 2023-05-09 桑格摩生物治疗股份有限公司 用于治疗遗传病状的方法和组合物
CA2920899C (en) 2013-08-28 2023-02-28 Sangamo Biosciences, Inc. Compositions for linking dna-binding domains and cleavage domains
ES2881473T3 (es) * 2013-10-17 2021-11-29 Sangamo Therapeutics Inc Métodos de suministro y composiciones para la modificación por ingeniería genética del genoma mediada por nucleasas
CA2926078C (en) 2013-10-17 2021-11-16 Sangamo Biosciences, 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
EP3960856A1 (en) 2013-11-13 2022-03-02 Children's Medical Center Corporation Nuclease-mediated regulation of gene expression
EP3080143B1 (en) 2013-12-09 2019-02-20 Sangamo Therapeutics, Inc. Methods and compositions for treating hemophilia
KR102170502B1 (ko) 2013-12-11 2020-10-28 리제너론 파마슈티칼스 인코포레이티드 게놈의 표적화된 변형을 위한 방법 및 조성물
ES2796087T3 (es) 2014-02-03 2020-11-25 Sangamo Therapeutics Inc Métodos y composiciones para el tratamiento de beta talasemia
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
WO2015143177A1 (en) 2014-03-21 2015-09-24 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
US11110154B2 (en) 2014-05-08 2021-09-07 Sangamo Therapeutics, Inc. Methods and compositions for treating Huntington's Disease
WO2015175642A2 (en) 2014-05-13 2015-11-19 Sangamo Biosciences, Inc. Methods and compositions for prevention or treatment of a disease
EP3151846A4 (en) 2014-06-05 2017-12-27 Sangamo BioSciences, Inc. Methods and compositions for nuclease design
US10106820B2 (en) 2014-06-06 2018-10-23 Regeneron Pharmaceuticals, Inc. Methods and compositions for modifying a targeted locus
HUE041584T2 (hu) 2014-06-26 2019-05-28 Regeneron Pharma Célzott genetikai módosítások és alkalmazási módszerek és készítmények
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
HRP20211468T1 (hr) 2014-09-16 2021-12-24 Sangamo Therapeutics, Inc. Postupci i pripravci za nukleazno posredovani inženjering genoma i ispravak u krvotvornim matičnim stanicama
RU2706965C2 (ru) 2014-10-15 2019-11-21 Регенерон Фармасьютикалз, Инк. Способы и композиции для получения или поддержания плюрипотентных клеток
JP6727199B2 (ja) 2014-11-21 2020-07-22 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. ガイドrnaのペアを使用したターゲティングによる遺伝子改変の方法及び組成物
US10889834B2 (en) 2014-12-15 2021-01-12 Sangamo Therapeutics, Inc. Methods and compositions for enhancing targeted transgene integration
ES2760508T3 (es) 2014-12-19 2020-05-14 Regeneron Pharma Métodos y composiciones para la modificación genética dirigida a través de la transformación múltiple en una sola etapa
WO2016118726A2 (en) 2015-01-21 2016-07-28 Sangamo Biosciences, Inc. Methods and compositions for identification of highly specific nucleases
ES2777534T3 (es) 2015-02-13 2020-08-05 Inserm - Institut National De La Santé Et De La Rech Médicale Polipéptidos para la ingeniería de proteínas integrasas quiméricas y su uso en terapia génica
US10179918B2 (en) 2015-05-07 2019-01-15 Sangamo Therapeutics, Inc. Methods and compositions for increasing transgene activity
KR20170141217A (ko) 2015-05-12 2017-12-22 상가모 테라퓨틱스, 인코포레이티드 유전자 발현의 뉴클레아제-매개된 조절
US9957501B2 (en) 2015-06-18 2018-05-01 Sangamo Therapeutics, Inc. Nuclease-mediated regulation of gene expression
CN108024544B (zh) 2015-07-13 2022-04-29 桑格摩生物治疗股份有限公司 用于核酸酶介导的基因组工程化的递送方法及组合物
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
EA037993B1 (ru) 2015-09-23 2021-06-21 Сангамо Терапьютикс, Инк. Репрессоры htt и их применение
PE20181206A1 (es) 2015-10-28 2018-07-23 Sangamo Therapeutics Inc Construcciones especificas del higado, casetes de expresion del factor viii y metodos de uso de estos
US10639383B2 (en) 2015-11-23 2020-05-05 Sangamo Therapeutics, Inc. Methods and compositions for engineering immunity
SG10202005640UA (en) 2015-12-18 2020-07-29 Sangamo Therapeutics Inc Targeted disruption of the mhc cell receptor
US11352631B2 (en) 2015-12-18 2022-06-07 Sangamo Therapeutics, Inc. Targeted disruption of the T cell receptor
ES2895430T3 (es) 2016-01-15 2022-02-21 Univ Minnesota Métodos y composiciones para el tratamiento de enfermedad neurológica
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
CN110325635B (zh) 2016-08-24 2023-12-26 桑格摩生物治疗股份有限公司 使用工程化的核酸酶调控基因表达
SG10202007392SA (en) 2016-08-24 2020-09-29 Sangamo Therapeutics Inc Engineered target specific nucleases
CA3035534A1 (en) 2016-09-07 2018-03-15 Sangamo Therapeutics, Inc. Modulation of liver genes
BR112019007210A2 (pt) 2016-10-20 2019-08-13 Sangamo Therapeutics Inc métodos e composições para o tratamento da doença de fabry
AU2017347928A1 (en) 2016-10-31 2019-05-09 Sangamo Therapeutics, Inc. Gene correction of scid-related genes in hematopoietic stem and progenitor cells
WO2018148462A1 (en) * 2017-02-09 2018-08-16 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
WO2018200943A1 (en) 2017-04-28 2018-11-01 Acuitas Therapeutics, Inc. Novel carbonyl lipids and lipid nanoparticle formulations for delivery of nucleic acids
CA3059793A1 (en) 2017-05-03 2018-11-08 Sangamo Therapeutics, Inc. Methods and compositions for modification of a cystic fibrosis transmembrane conductance regulator (cftr) gene
US11512287B2 (en) 2017-06-16 2022-11-29 Sangamo Therapeutics, Inc. Targeted disruption of T cell and/or HLA receptors
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
KR20200033259A (ko) 2017-07-31 2020-03-27 리제너론 파마슈티칼스 인코포레이티드 생체내에서 CRISPR/Cas-매개된 파괴 또는 삭제 및 외인성 도너 핵산과의 CRISPR/Cas-유도된 재조합을 평가하기 위한 방법 및 조성물
CN110891420B (zh) 2017-07-31 2022-06-03 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
AU2018364660B2 (en) 2017-11-09 2022-05-19 Sangamo Therapeutics, Inc. Genetic modification of cytokine inducible SH2-containing protein (CISH) gene
BR112020015488A2 (pt) 2018-02-08 2020-12-08 Sangamo Therapeutics, Inc. Nucleases alvo-específicas modificadas
SG11202007697VA (en) 2018-02-15 2020-09-29 Memorial Sloan Kettering Cancer Center Foxp3 targeting agent compositions and methods of use for adoptive cell therapy
KR102647714B1 (ko) 2018-03-19 2024-03-18 리제너론 파마슈티칼스 인코포레이티드 CRISPR/Cas 시스템을 사용한 동물에서의 전사 조절
RU2020136054A (ru) 2018-04-05 2022-05-06 Джуно Терапьютикс, Инк. Т-клетки, экспрессирующие рекомбинантный рецептор, соответствующие полинуклеотиды и способы
BR112020020245A2 (pt) 2018-04-05 2021-04-06 Editas Medicine, Inc. Métodos de produzir células expressando um receptor recombinante e composições relacionadas
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
SG11202101801RA (en) 2018-08-23 2021-03-30 Sangamo Therapeutics Inc Engineered target specific base editors
US20220098613A1 (en) 2018-09-12 2022-03-31 Fred Hutchinson Cancer Research Center Reducing cd33 expression to selectively protect therapeutic cells
CN113015741A (zh) 2018-09-18 2021-06-22 桑格摩生物治疗股份有限公司 程序性细胞死亡1(pd1)特异性核酸酶
WO2020069029A1 (en) 2018-09-26 2020-04-02 Emendobio Inc. Novel crispr nucleases
MA53919A (fr) 2018-10-18 2021-08-25 Intellia Therapeutics Inc Constructions d'acides nucléiques et procédés d'utilisation
EP3886869A4 (en) 2018-11-28 2022-07-06 Forty Seven, Inc. GENETICALLY MODIFIED CSPH RESISTANT TO ABLATIVE TREATMENT
CN113474328A (zh) 2019-01-11 2021-10-01 爱康泰生治疗公司 用于脂质纳米颗粒递送活性剂的脂质
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
AU2020256225A1 (en) 2019-04-03 2021-09-02 Regeneron Pharmaceuticals, Inc. Methods and compositions for insertion of antibody coding sequences into a safe harbor locus
MX2021013219A (es) 2019-05-01 2022-02-17 Juno Therapeutics Inc Celulas que expresan un receptor recombinante de un locus de tgfbr2 modificado, polinucleotidos relacionados y metodos.
CA3136742A1 (en) 2019-05-01 2020-11-05 Juno Therapeutics, Inc. Cells expressing a chimeric receptor from a modified cd247 locus, related polynucleotides and methods
CN114729383A (zh) 2019-07-02 2022-07-08 弗莱德哈钦森癌症研究中心 重组ad35载体及相关基因疗法改进
WO2021072115A1 (en) 2019-10-08 2021-04-15 Regents Of The University Of Minnesota Crispr-mediated human genome editing with vectors
IL292605A (en) 2019-11-08 2022-07-01 Regeneron Pharma CRISPR and AAV strategies for the treatment of x-linked childhood retinoschisis
WO2021108363A1 (en) 2019-11-25 2021-06-03 Regeneron Pharmaceuticals, Inc. Crispr/cas-mediated upregulation of humanized ttr allele
EP4150057A2 (en) 2020-05-13 2023-03-22 Juno Therapeutics, Inc. Process for producing donor-batched cells expressing a recombinant receptor
US20230183750A1 (en) 2020-05-21 2023-06-15 Oxford Genetics Limited Hdr enhancers
WO2021234389A1 (en) 2020-05-21 2021-11-25 Oxford Genetics Limited Hdr enhancers
GB202007578D0 (en) 2020-05-21 2020-07-08 Univ Oxford Innovation Ltd Hdr enhancers
GB202007577D0 (en) 2020-05-21 2020-07-08 Oxford Genetics Ltd Hdr enhancers
US20240216508A1 (en) 2020-06-26 2024-07-04 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
KR20230051172A (ko) 2020-07-16 2023-04-17 아퀴타스 테라퓨틱스 인크. 지질 나노 입자에 사용하기 위한 양이온성 지질
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
EP4313122A1 (en) 2021-03-23 2024-02-07 Iovance Biotherapeutics, Inc. Cish gene editing of tumor infiltrating lymphocytes and uses of same in immunotherapy
WO2023077012A1 (en) 2021-10-27 2023-05-04 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
WO2023108047A1 (en) 2021-12-08 2023-06-15 Regeneron Pharmaceuticals, Inc. Mutant myocilin disease model and uses thereof
TW202332767A (zh) 2022-02-02 2023-08-16 美商雷傑納榮製藥公司 用於治療龐貝氏症之抗TfR:GAA及抗CD63:GAA插入
WO2023150393A2 (en) 2022-02-07 2023-08-10 Ensoma, Inc. Inhibitor-resistant mgmt modifications and modification of mgmt-encoding nucleic acids
WO2023150798A1 (en) 2022-02-07 2023-08-10 Regeneron Pharmaceuticals, Inc. Compositions and methods for defining optimal treatment timeframes in lysosomal disease
WO2023220043A1 (en) 2022-05-09 2023-11-16 Synteny Therapeutics, Inc. Erythroparvovirus with a modified genome for gene therapy
WO2023220040A1 (en) 2022-05-09 2023-11-16 Synteny Therapeutics, Inc. Erythroparvovirus with a modified capsid for gene therapy
WO2023220035A1 (en) 2022-05-09 2023-11-16 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
WO2024073606A1 (en) 2022-09-28 2024-04-04 Regeneron Pharmaceuticals, Inc. Antibody resistant modified receptors to enhance cell-based therapies
WO2024098002A1 (en) 2022-11-04 2024-05-10 Regeneron Pharmaceuticals, Inc. Calcium voltage-gated channel auxiliary subunit gamma 1 (cacng1) binding proteins and cacng1-mediated delivery to skeletal muscle
WO2024100604A1 (en) 2022-11-09 2024-05-16 Juno Therapeutics Gmbh Methods for manufacturing engineered immune cells
WO2024107765A2 (en) 2022-11-14 2024-05-23 Regeneron Pharmaceuticals, Inc. Compositions and methods for fibroblast growth factor receptor 3-mediated delivery to astrocytes

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
EP0770129B1 (en) 1994-01-18 2005-11-23 The Scripps Research Institute Zinc finger protein derivatives and methods therefor
US6140466A (en) 1994-01-18 2000-10-31 The Scripps Research Institute Zinc finger protein derivatives and methods therefor
US6242568B1 (en) * 1994-01-18 2001-06-05 The Scripps Research Institute Zinc finger protein derivatives and methods therefor
GB9824544D0 (en) 1998-11-09 1999-01-06 Medical Res Council Screening system
US6007988A (en) 1994-08-20 1999-12-28 Medical Research Council Binding proteins for recognition of DNA
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
AU746454B2 (en) * 1998-03-02 2002-05-02 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
US6453242B1 (en) * 1999-01-12 2002-09-17 Sangamo Biosciences, Inc. Selection of sites for targeting by zinc finger proteins and methods of designing zinc finger proteins to bind to preselected sites
US6534261B1 (en) * 1999-01-12 2003-03-18 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
US6794136B1 (en) 2000-11-20 2004-09-21 Sangamo Biosciences, Inc. Iterative optimization in the design of binding proteins
ATE355368T1 (de) 2000-01-24 2006-03-15 Gendaq Ltd Nucleinsäure bindende polypeptide gekennzeichnet durch flexible linker verbundene nucleinsäuredomäne
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
US7273923B2 (en) 2001-01-22 2007-09-25 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
EP1421177A4 (en) 2001-08-20 2006-06-07 Scripps Research Inst ZINC FINGER FASTENING DOMAINS FOR CNN
DE60310202T2 (de) * 2002-01-23 2007-11-22 The University Of Utah Research Foundation, Salt Lake City Zielgerichtete chromosomale mutagenese mit zinkfingernukleasen
US20030232410A1 (en) * 2002-03-21 2003-12-18 Monika Liljedahl 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
KR20060039019A (ko) * 2003-08-08 2006-05-04 상가모 바이오사이언스 인코포레이티드 표적화된 절단과 재조합을 위한 방법 및 그 조성물
US7888121B2 (en) * 2003-08-08 2011-02-15 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
US20080131962A1 (en) 2006-05-25 2008-06-05 Sangamo Biosciences, Inc. Engineered cleavage half-domains
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
CA2615532C (en) 2005-07-26 2016-06-28 Sangamo Biosciences, Inc. Targeted integration and expression of exogenous nucleic acid sequences

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