ATE536371T1 - Verfahren und zusammensetzungen zur gendeaktivierung - Google Patents

Verfahren und zusammensetzungen zur gendeaktivierung

Info

Publication number
ATE536371T1
ATE536371T1 AT07795401T AT07795401T ATE536371T1 AT E536371 T1 ATE536371 T1 AT E536371T1 AT 07795401 T AT07795401 T AT 07795401T AT 07795401 T AT07795401 T AT 07795401T AT E536371 T1 ATE536371 T1 AT E536371T1
Authority
AT
Austria
Prior art keywords
zfns
compositions
polynucleotides encoding
vectors
cells
Prior art date
Application number
AT07795401T
Other languages
English (en)
Inventor
Dale Ando
Michael Christopher Holmes
Gary Ka Leong Lee
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 ATE536371T1 publication Critical patent/ATE536371T1/de

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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
    • 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]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00—Cellular immunotherapy
    • A61K40/10—Cellular immunotherapy characterised by the cell type used
    • A61K40/11—T-cells, e.g. tumour infiltrating lymphocytes [TIL] or regulatory T [Treg] cells; Lymphokine-activated killer [LAK] cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00—Cellular immunotherapy
    • A61K40/40—Cellular immunotherapy characterised by antigens that are targeted or presented by cells of the immune system
    • A61K40/46—Viral antigens
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A61P31/14—Antivirals for RNA viruses
    • A61P31/18—Antivirals for RNA viruses for HIV
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • 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
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • 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
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4702—Regulators; Modulating activity
    • C07K14/4703—Inhibitors; Suppressors
    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—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
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/10—Cells modified by introduction of foreign genetic material
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K2035/124—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells the cells being hematopoietic, bone marrow derived or blood cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2239/00—Indexing codes associated with cellular immunotherapy of group A61K40/00
    • A61K2239/31—Indexing codes associated with cellular immunotherapy of group A61K40/00 characterized by the route of administration
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2239/00—Indexing codes associated with cellular immunotherapy of group A61K40/00
    • A61K2239/38—Indexing codes associated with cellular immunotherapy of group A61K40/00 characterised by the dose, timing or administration schedule
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • 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
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/10011—Adenoviridae
    • C12N2710/10311—Mastadenovirus, e.g. human or simian adenoviruses
    • C12N2710/10341—Use of virus, viral particle or viral elements as a vector
    • C12N2710/10343—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • 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
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/10011—Adenoviridae
    • C12N2710/10311—Mastadenovirus, e.g. human or simian adenoviruses
    • C12N2710/10341—Use of virus, viral particle or viral elements as a vector
    • C12N2710/10345—Special targeting system for viral vectors

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • General Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Cell Biology (AREA)
  • Immunology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Physics & Mathematics (AREA)
  • Developmental Biology & Embryology (AREA)
  • Plant Pathology (AREA)
  • Communicable Diseases (AREA)
  • AIDS & HIV (AREA)
  • Tropical Medicine & Parasitology (AREA)
  • Oncology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
AT07795401T 2006-05-25 2007-05-23 Verfahren und zusammensetzungen zur gendeaktivierung ATE536371T1 (de)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US80850106P 2006-05-25 2006-05-25
US84726906P 2006-09-26 2006-09-26
US92691107P 2007-04-30 2007-04-30
PCT/US2007/012588 WO2007139982A2 (en) 2006-05-25 2007-05-23 Methods and compositions for gene inactivation

Publications (1)

Publication Number Publication Date
ATE536371T1 true ATE536371T1 (de) 2011-12-15

Family

ID=38779263

Family Applications (1)

Application Number Title Priority Date Filing Date
AT07795401T ATE536371T1 (de) 2006-05-25 2007-05-23 Verfahren und zusammensetzungen zur gendeaktivierung

Country Status (8)

Country Link
US (7) US7951925B2 (de)
EP (4) EP2019839B1 (de)
JP (2) JP5551432B2 (de)
AT (1) ATE536371T1 (de)
AU (1) AU2007267874B2 (de)
CA (1) CA2651499C (de)
ES (2) ES2465996T3 (de)
WO (1) WO2007139982A2 (de)

Families Citing this family (233)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US8278079B2 (en) * 2002-11-07 2012-10-02 Danisco Us Inc. BGL6 β-glucosidase and nucleic acids encoding the same
US20120196370A1 (en) 2010-12-03 2012-08-02 Fyodor Urnov Methods and compositions for targeted genomic deletion
EP2019839B1 (de) 2006-05-25 2011-12-07 Sangamo BioSciences, Inc. Verfahren und zusammensetzungen zur gendeaktivierung
US9217026B2 (en) 2006-11-13 2015-12-22 Sangamo Biosciences, Inc. Method of inactivating a glucocorticoid receptor gene in an isolated cell
JP5666903B2 (ja) * 2007-05-23 2015-02-12 サンガモ バイオサイエンシーズ， インコーポレイテッド 導入遺伝子の発現を増強するための方法および組成物
US8563314B2 (en) 2007-09-27 2013-10-22 Sangamo Biosciences, Inc. Methods and compositions for modulating PD1
US11235026B2 (en) 2007-09-27 2022-02-01 Sangamo Therapeutics, Inc. Methods and compositions for modulating PD1
CN101883863B (zh) * 2007-09-27 2018-01-23 桑格摩生物科学股份有限公司 生物活性核酸酶的快速体内鉴定
CA2703045C (en) 2007-10-25 2017-02-14 Sangamo Biosciences, Inc. Methods and compositions for targeted integration
ES2551318T3 (es) 2008-04-21 2015-11-18 Danziger Innovations Ltd. Vectores de expresión virales de plantas y uso de los mismos para generar variaciones genotípicas en genomas de plantas
JP2011521643A (ja) 2008-05-28 2011-07-28 サンガモ バイオサイエンシーズ， インコーポレイテッド Ｄｎａ結合ドメインおよび切断ドメインを連結するための組成物
JP5763530B2 (ja) * 2008-06-10 2015-08-12 サンガモ バイオサイエンシーズ， インコーポレイテッド Ｂａｘ−およびＢａｋ−欠損細胞株の生成のための方法および組成物
SG191561A1 (en) * 2008-08-22 2013-07-31 Sangamo Biosciences Inc Methods and compositions for targeted single-stranded cleavage and targeted integration
CN102264915B (zh) 2008-10-29 2014-04-16 桑格摩生物科学股份有限公司 失活谷氨酰胺合成酶基因表达的方法和组合物
US20110016540A1 (en) * 2008-12-04 2011-01-20 Sigma-Aldrich Co. Genome editing of genes associated with trinucleotide repeat expansion disorders in animals
US20110023140A1 (en) * 2008-12-04 2011-01-27 Sigma-Aldrich Co. Rabbit genome editing with zinc finger nucleases
US20110023139A1 (en) * 2008-12-04 2011-01-27 Sigma-Aldrich Co. Genomic editing of genes involved in cardiovascular disease
US20110023154A1 (en) * 2008-12-04 2011-01-27 Sigma-Aldrich Co. Silkworm genome editing with zinc finger nucleases
US20110023150A1 (en) * 2008-12-04 2011-01-27 Sigma-Aldrich Co. Genome editing of genes associated with schizophrenia in animals
US20110023145A1 (en) * 2008-12-04 2011-01-27 Sigma-Aldrich Co. Genomic editing of genes involved in autism spectrum disorders
US20110023149A1 (en) * 2008-12-04 2011-01-27 Sigma-Aldrich Co. Genomic editing of genes involved in tumor suppression in animals
US20110023146A1 (en) * 2008-12-04 2011-01-27 Sigma-Aldrich Co. Genomic editing of genes involved in secretase-associated disorders
US20110023158A1 (en) * 2008-12-04 2011-01-27 Sigma-Aldrich Co. Bovine genome editing with zinc finger nucleases
US20110016546A1 (en) * 2008-12-04 2011-01-20 Sigma-Aldrich Co. Porcine genome editing with zinc finger nucleases
US20110016541A1 (en) * 2008-12-04 2011-01-20 Sigma-Aldrich Co. Genome editing of sensory-related genes in animals
US20110023153A1 (en) * 2008-12-04 2011-01-27 Sigma-Aldrich Co. Genomic editing of genes involved in alzheimer's disease
US20110016543A1 (en) * 2008-12-04 2011-01-20 Sigma-Aldrich Co. Genomic editing of genes involved in inflammation
US20110023143A1 (en) * 2008-12-04 2011-01-27 Sigma-Aldrich Co. Genomic editing of neurodevelopmental genes in animals
US20110023141A1 (en) * 2008-12-04 2011-01-27 Sigma-Aldrich Co. Genomic editing of genes involved with parkinson's disease
US20110023144A1 (en) * 2008-12-04 2011-01-27 Sigma-Aldrich Co. Genomic editing of genes involved in amyotrophyic lateral sclerosis disease
JP5681114B2 (ja) 2008-12-04 2015-03-04 サンガモ バイオサイエンシーズ， インコーポレイテッド 亜鉛フィンガーヌクレアーゼを使用したラットのゲノム編集
US20110023147A1 (en) * 2008-12-04 2011-01-27 Sigma-Aldrich Co. Genomic editing of prion disorder-related genes in animals
US20110023152A1 (en) * 2008-12-04 2011-01-27 Sigma-Aldrich Co. Genome editing of cognition related genes in animals
US20110023151A1 (en) * 2008-12-04 2011-01-27 Sigma-Aldrich Co. Genome editing of abc transporters
US20110023148A1 (en) * 2008-12-04 2011-01-27 Sigma-Aldrich Co. Genome editing of addiction-related genes in animals
US20110016539A1 (en) * 2008-12-04 2011-01-20 Sigma-Aldrich Co. Genome editing of neurotransmission-related genes in animals
US20110023156A1 (en) * 2008-12-04 2011-01-27 Sigma-Aldrich Co. Feline genome editing with zinc finger nucleases
US20110030072A1 (en) * 2008-12-04 2011-02-03 Sigma-Aldrich Co. Genome editing of immunodeficiency genes in animals
AU2010226313B2 (en) * 2009-03-20 2014-10-09 Sangamo Therapeutics, Inc. Modification of CXCR4 using engineered zinc finger proteins
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
WO2011002503A1 (en) 2009-06-30 2011-01-06 Sangamo Biosciences, Inc. Rapid screening of biologically active nucleases and isolation of nuclease-modified cells
US9234016B2 (en) * 2009-07-28 2016-01-12 Sangamo Biosciences, Inc. Engineered zinc finger proteins for treating trinucleotide repeat disorders
US9476060B2 (en) 2009-10-21 2016-10-25 Danziger Innovations Ltd. Generating genotypic variations in plant genomes by gamete infection
US8956828B2 (en) * 2009-11-10 2015-02-17 Sangamo Biosciences, Inc. Targeted disruption of T cell receptor genes using engineered zinc finger protein nucleases
WO2011064750A1 (en) * 2009-11-27 2011-06-03 Basf Plant Science Company Gmbh Chimeric endonucleases and uses thereof
WO2011086118A1 (en) * 2010-01-14 2011-07-21 Deutsches Krebsforschungszentrum In vivo determination of the lokalization of dna double-stand breaks and application thereof
NZ601247A (en) * 2010-01-22 2014-10-31 Dow Agrosciences Llc Targeted genomic alteration
DK2534173T3 (da) 2010-02-08 2019-10-14 Sangamo Therapeutics Inc Manipulerede spaltnings-halvdomæner
EP2534163B1 (de) * 2010-02-09 2015-11-04 Sangamo BioSciences, Inc. Gezielte genommodifikation mit partiell einsträngigen donatormolekülen
KR101880536B1 (ko) 2010-04-26 2018-07-23 상가모 테라퓨틱스, 인코포레이티드 아연 핑거 뉴클레아제를 사용하는 로사 좌위의 게놈 편집
KR101974036B1 (ko) 2010-05-03 2019-04-30 상가모 테라퓨틱스, 인코포레이티드 아연 핑거 모듈을 연결하기 위한 조성물
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
EP2580331A4 (de) * 2010-06-14 2013-11-27 Univ Iowa State Res Found Inc Nukleaseaktivität des tal-effektors und des fusionsproteins foki
CA2993567C (en) 2010-07-21 2022-06-28 Sangamo Biosciences, Inc. Methods and compositions for modification of a t-cell receptor gene
JP2013537410A (ja) 2010-07-23 2013-10-03 シグマ−アルドリッチ・カンパニー・リミテッド・ライアビリティ・カンパニー 標的化エンドヌクレアーゼおよび一本鎖核酸を用いたゲノム編集
WO2012015938A2 (en) * 2010-07-27 2012-02-02 The Johns Hopkins University Obligate heterodimer variants of foki cleavage domain
WO2012018726A1 (en) * 2010-08-02 2012-02-09 Cellectis Sa Method for increasing double-strand break-induced gene targeting
CA2811364C (en) 2010-09-27 2022-01-04 Sangamo Biosciences, Inc. Methods and compositions for inhibiting viral entry into cells
CN103270046B (zh) 2010-10-12 2016-05-11 费城儿童医院 治疗b型血友病的方法和组合物
EP2441774B1 (de) 2010-10-15 2018-12-12 Fundació Centre de Regulació Genòmica Peptide mit Zink Finger Domänen und deren Verwendungen
CN102234628A (zh) * 2011-05-06 2011-11-09 中国科学院广州生物医药与健康研究院 Ccr5缺失型的造血干细胞及其制备方法与应用
EP2522726A1 (de) 2011-05-12 2012-11-14 Fundació Privada Centre de Regulació Genòmica (CRG) Zinkfingernukleasen für p53-Änderung
CA2848417C (en) 2011-09-21 2023-05-02 Sangamo Biosciences, Inc. Methods and compositions for regulation of transgene expression
EP2758537A4 (de) * 2011-09-23 2015-08-12 Univ Iowa State Res Found Monomerarchitektur aus tal-nuklease oder zinkfingernuklease zur dna-modifikation
CA2852955C (en) 2011-10-27 2021-02-16 Sangamo Biosciences, Inc. Methods and compositions for modification of the hprt locus
JP6144691B2 (ja) 2011-11-16 2017-06-07 サンガモ セラピューティクス， インコーポレイテッド 修飾されたｄｎａ結合タンパク質およびその使用
US9688997B2 (en) 2011-12-29 2017-06-27 Iowa State University Research Foundation, Inc. Genetically modified plants with resistance to Xanthomonas and other bacterial plant pathogens
GB201122458D0 (en) 2011-12-30 2012-02-08 Univ Wageningen Modified cascade ribonucleoproteins and uses thereof
NZ629427A (en) 2012-02-29 2016-04-29 Sangamo Biosciences Inc Methods and compositions for treating huntington’s disease
CA2871524C (en) 2012-05-07 2021-07-27 Sangamo Biosciences, Inc. Methods and compositions for nuclease-mediated targeted integration of transgenes
PL4289948T3 (pl) 2012-05-25 2025-06-02 The Regents Of The University Of California Sposoby i kompozycje do modyfikacji kierowanego na rna docelowego dna i do nakierowanej na rna modulacji transkrypcji
US10648001B2 (en) 2012-07-11 2020-05-12 Sangamo Therapeutics, Inc. Method of treating mucopolysaccharidosis type I or II
HUE051612T2 (hu) 2012-07-11 2021-03-01 Sangamo Therapeutics Inc Eljárások és készítmények lizoszomális tárolási betegségek kezelésére
EP2872154B1 (de) 2012-07-11 2017-05-31 Sangamo BioSciences, Inc. Verfahren und zusammensetzungen zur freisetzung von biologischen wirkstoffen
US20150104873A1 (en) * 2012-07-11 2015-04-16 University of Nevada, Las Vegas Genome Surgery with Paired, Permeant Endonuclease Construct
PL2890780T3 (pl) 2012-08-29 2021-02-08 Sangamo Therapeutics, Inc. Sposoby i kompozycje do leczenia zaburzeń genetycznych
AU2013329186B2 (en) 2012-10-10 2019-02-14 Sangamo Therapeutics, Inc. T cell modifying compounds and uses thereof
US20140140969A1 (en) * 2012-11-20 2014-05-22 Sangamo Biosciences, Inc. Methods and compositions for muscular dystrophies
CA2892448A1 (en) 2012-12-05 2014-06-12 Sangamo Biosciences, Inc. Methods and compositions for regulation of metabolic disorders
CN105142669B (zh) 2012-12-06 2018-07-03 西格马-奥尔德里奇有限责任公司 基于crispr的基因组修饰和调控
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
KR20170108172A (ko) 2013-03-14 2017-09-26 카리부 바이오사이언시스 인코포레이티드 핵산-표적화 핵산의 조성물 및 방법
WO2014153470A2 (en) 2013-03-21 2014-09-25 Sangamo Biosciences, Inc. Targeted disruption of t cell receptor genes using engineered zinc finger protein nucleases
JP2016522679A (ja) * 2013-04-04 2016-08-04 プレジデント アンド フェローズ オブ ハーバード カレッジ ＣＲＩＳＰＲ／Ｃａｓ系を用いたゲノム編集の治療的使用
US10604771B2 (en) * 2013-05-10 2020-03-31 Sangamo Therapeutics, 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
CA2926078C (en) 2013-10-17 2021-11-16 Sangamo Biosciences, Inc. Delivery methods and compositions for nuclease-mediated genome engineering in hematopoietic stem cells
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
NZ719477A (en) 2013-11-11 2022-05-27 Sangamo Therapeutics Inc Methods and compositions for treating huntington’s disease
PL3068881T3 (pl) 2013-11-13 2019-08-30 Children's Medical Center Corporation Regulacja ekspresji genów, w której pośredniczą nukleazy
WO2015071474A2 (en) * 2013-11-18 2015-05-21 Crispr Therapeutics Ag Crispr-cas system materials and methods
EP3080274B1 (de) 2013-12-09 2020-06-03 Sangamo Therapeutics, Inc. Verfahren und zusammensetzungen für genom-engineering
SI3102673T1 (sl) 2014-02-03 2020-11-30 Sangamo Therapeutics, Inc. Postopki in sestavki za zdravljenje beta talasemije
AU2015218576B2 (en) 2014-02-24 2020-02-27 Sangamo Therapeutics, Inc. Methods and compositions for nuclease-mediated targeted integration
KR101576728B1 (ko) * 2014-03-06 2015-12-10 가톨릭대학교 산학협력단 면역세포 백신 및 이의 용도
ES2879373T3 (es) 2014-03-18 2021-11-22 Sangamo Therapeutics Inc Métodos y composiciones para la regulación de la expresión de proteínas de dedo de zinc
US9522936B2 (en) 2014-04-24 2016-12-20 Sangamo Biosciences, Inc. Engineered transcription activator like effector (TALE) proteins
MX378789B (es) 2014-05-08 2025-03-11 Sangamo Biosciences Inc Métodos y composiciones para tratar la enfermedad de huntington.
AU2015259191B2 (en) 2014-05-13 2019-03-21 Sangamo Therapeutics, Inc. Methods and compositions for prevention or treatment of a disease
EP3152319A4 (de) 2014-06-05 2017-12-27 Sangamo BioSciences, Inc. Verfahren und zusammensetzungen zur nukleasekonstruktion
US9816074B2 (en) 2014-07-25 2017-11-14 Sangamo Therapeutics, Inc. Methods and compositions for modulating nuclease-mediated genome engineering in hematopoietic stem cells
US9757420B2 (en) 2014-07-25 2017-09-12 Sangamo Therapeutics, 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
WO2016037161A2 (en) 2014-09-07 2016-03-10 Selecta Biosciences, Inc. Methods and compositions for attenuating gene editing anti-viral transfer vector immune responses
CN113699113A (zh) 2014-09-16 2021-11-26 桑格摩治疗股份有限公司 用于造血干细胞中核酸酶介导的基因组工程化和校正的方法和组合物
CN112553229A (zh) 2014-11-05 2021-03-26 沃雅戈治疗公司 用于治疗帕金森病的aadc多核苷酸
RU2020108189A (ru) 2014-11-14 2020-03-11 Вояджер Терапьютикс, Инк. Композиции и способы лечения бокового амиотрофического склероза (als)
MX2017006217A (es) 2014-11-14 2018-05-02 Voyager Therapeutics Inc Polinucleotidos moduladores.
EP3025719B1 (de) 2014-11-26 2018-09-26 Miltenyi Biotec GmbH Kombinationsimmuntherapie für antigen-erkennende rezeptoren und hämatopoetische zellen zur behandlung von krankheiten
WO2016084084A1 (en) 2014-11-27 2016-06-02 Danziger Innovations Ltd. Nucleic acid constructs for genome editing
US10889834B2 (en) 2014-12-15 2021-01-12 Sangamo Therapeutics, Inc. Methods and compositions for enhancing targeted transgene integration
EP3247366A4 (de) 2015-01-21 2018-10-31 Sangamo Therapeutics, Inc. Verfahren und zusammensetzungen zur identifizierung hochspezifischer nukleasen
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
US20180080051A1 (en) 2015-03-31 2018-03-22 Exeligen Scientific, Inc. Cas 9 retroviral integrase and cas 9 recombinase systems for targeted incorporation of a dna sequence into a genome of a cell or organism
ES2959608T3 (es) * 2015-04-03 2024-02-27 Dana Farber Cancer Inst Inc Composición y métodos de edición del genoma de células B
US10179918B2 (en) 2015-05-07 2019-01-15 Sangamo Therapeutics, Inc. Methods and compositions for increasing transgene activity
CN107921148A (zh) 2015-05-08 2018-04-17 哈佛学院校长同事会 通用供体干细胞和相关方法
EP3294866A4 (de) * 2015-05-12 2018-12-05 Sangamo Therapeutics, Inc. Nukleasevermittelte regulierung der genexpression
EP3294879A4 (de) * 2015-05-14 2019-02-20 University of Southern California Optimierte geneditierung mit einem rekombinanten endonukleasesystem
US9957501B2 (en) 2015-06-18 2018-05-01 Sangamo Therapeutics, Inc. Nuclease-mediated regulation of gene expression
AU2016291778B2 (en) 2015-07-13 2021-05-06 Sangamo Therapeutics, Inc. Delivery methods and compositions for nuclease-mediated genome engineering
US10435441B2 (en) 2015-09-23 2019-10-08 Sangamo Therapeutics, Inc. HTT repressors and uses thereof
WO2017053729A1 (en) 2015-09-25 2017-03-30 The Board Of Trustees Of The Leland Stanford Junior University Nuclease-mediated genome editing of primary cells and enrichment thereof
KR102766526B1 (ko) 2015-10-28 2025-02-13 상가모 테라퓨틱스, 인코포레이티드 간-특이적 작제물인, 인자 viii 발현 카세트 및 이의 사용 방법
WO2017079428A1 (en) 2015-11-04 2017-05-11 President And Fellows Of Harvard College Site specific germline modification
JP6976249B2 (ja) 2015-11-23 2021-12-08 サンガモ セラピューティクス， インコーポレイテッド 免疫を工学操作するための方法および組成物
WO2017106528A2 (en) 2015-12-18 2017-06-22 Sangamo Biosciences, Inc. Targeted disruption of the t cell receptor
MX2018007519A (es) 2015-12-18 2019-09-04 Sangamo Therapeutics Inc Alteracion dirigida del receptor celular del complejo mayor de histocompatibilidad (mhc).
ES2895430T3 (es) 2016-01-15 2022-02-21 Univ Minnesota Métodos y composiciones para el tratamiento de enfermedad neurológica
JP7012650B2 (ja) 2016-02-02 2022-01-28 サンガモ セラピューティクス， インコーポレイテッド 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
AU2017267665C1 (en) 2016-05-18 2023-10-05 Voyager Therapeutics, Inc. Modulatory polynucleotides
BR112018073472A2 (pt) 2016-05-18 2019-08-27 Voyager Therapeutics, Inc. composições e métodos de tratamento da doença de huntington
CR20190098A (es) 2016-08-24 2019-04-30 Sangamo Therapeutics Inc Regulación de la expresión génica usando nucleasas modificadas
SG11201901364VA (en) 2016-08-24 2019-03-28 Sangamo Therapeutics Inc Engineered target specific nucleases
WO2018049009A2 (en) 2016-09-07 2018-03-15 Sangamo Therapeutics, Inc. Modulation of liver genes
JP7042263B2 (ja) 2016-10-20 2022-03-25 サンガモ セラピューティクス， インコーポレイテッド ファブリー病の治療のための方法および組成物
EP3532106A4 (de) 2016-10-31 2020-06-24 Sangamo Therapeutics, Inc. Genkorrektur von scid-assoziierten genen bei hämotopoietischen stammzellen und vorläuferzellen
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
CA3061652A1 (en) 2017-05-05 2018-11-08 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
CN111108198A (zh) 2017-05-05 2020-05-05 沃雅戈治疗公司 治疗亨廷顿病的组合物和方法
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
US11512287B2 (en) 2017-06-16 2022-11-29 Sangamo Therapeutics, Inc. Targeted disruption of T cell and/or HLA receptors
US12178830B2 (en) 2017-06-30 2024-12-31 Memorial Sloan Kettering Cancer Center Compositions and methods for adoptive cell therapy for cancer
EP3585160A2 (de) 2017-07-31 2020-01-01 Regeneron Pharmaceuticals, Inc. Nichtmenschliche crispr-reporter-tiere und verwendungen davon
SG11201912235PA (en) 2017-07-31 2020-01-30 Regeneron Pharma Cas-transgenic mouse embryonic stem cells and mice and uses thereof
AU2018309714A1 (en) 2017-07-31 2020-01-30 Regeneron Pharmaceuticals, Inc. Assessment of CRISPR/Cas-induced recombination with an exogenous donor nucleic acid in vivo
WO2019032675A1 (en) 2017-08-08 2019-02-14 Sangamo Therapeutics, Inc. CELL TARGETING MEDIATED BY A CHIMERIC ANTIGEN RECEPTOR
AU2018347583A1 (en) 2017-10-13 2020-05-21 Selecta Biosciences, Inc. Methods and compositions for attenuating anti-viral transfer vector IgM responses
JP2021502085A (ja) 2017-11-09 2021-01-28 サンガモ セラピューティクス， インコーポレイテッド サイトカイン誘導性ｓｈ２含有タンパク質（ｃｉｓｈ）遺伝子の遺伝子改変
MX2020008272A (es) 2018-02-08 2020-09-21 Sangamo Therapeutics Inc Nucleasas diseñadas específicas de blancos.
WO2019161133A1 (en) 2018-02-15 2019-08-22 Memorial Sloan Kettering Cancer Center Foxp3 targeting agent compositions and methods of use for adoptive cell therapy
US12178908B2 (en) 2018-02-26 2024-12-31 AnTolRx, Inc. Tolerogenic liposomes and methods of use thereof
KR20240038811A (ko) 2018-03-19 2024-03-25 리제너론 파마슈티칼스 인코포레이티드 CRISPR/Cas 시스템을 사용한 동물에서의 전사 조절
EP3775237A1 (de) 2018-04-05 2021-02-17 Juno Therapeutics, Inc. T-zellen, die einen rekombinanten rezeptor exprimieren, verwandte polynukleotide und verfahren
KR20210029707A (ko) 2018-04-05 2021-03-16 주노 쎄러퓨티크스 인코퍼레이티드 재조합 수용체를 발현하는 세포의 생산 방법 및 관련 조성물
WO2019204457A1 (en) 2018-04-18 2019-10-24 Sangamo Therapeutics, Inc. Zinc finger protein compositions for modulation of huntingtin (htt)
AU2019268330A1 (en) 2018-05-15 2020-11-26 Voyager Therapeutics, Inc. Compositions and methods for the treatment of Parkinson's disease
US11690921B2 (en) 2018-05-18 2023-07-04 Sangamo Therapeutics, Inc. Delivery of target specific nucleases
KR102712986B1 (ko) 2018-08-23 2024-10-07 상가모 테라퓨틱스, 인코포레이티드 조작된 표적 특이적 염기 편집기
EP3849565A4 (de) 2018-09-12 2022-12-28 Fred Hutchinson Cancer Research Center Verminderung der cd33-expression zum selektiven schutz von therapeutischen zellen
AU2019344927A1 (en) 2018-09-18 2021-04-01 Sangamo Therapeutics, Inc. Programmed cell death 1 (PD1) specific nucleases
EP4613367A1 (de) 2018-09-21 2025-09-10 Acuitas Therapeutics, Inc. Systeme und verfahren zur herstellung von lipidnanopartikeln und liposomen
WO2020069029A1 (en) 2018-09-26 2020-04-02 Emendobio Inc. Novel crispr nucleases
JP7633155B2 (ja) * 2018-10-11 2025-02-19 オスペダーレ サン ラファエレ エス．アール．エル 人工トランス活性化因子による選択
EP3867380A2 (de) 2018-10-18 2021-08-25 Intellia Therapeutics, Inc. Zusammensetzungen und verfahren zur expression von faktor ix
AU2019360269A1 (en) 2018-10-18 2021-05-27 Intellia Therapeutics, Inc. Nucleic acid constructs and methods of use
AU2019390394C1 (en) 2018-11-28 2024-11-28 Forty Seven, LLC Genetically modified HSPCs resistant to ablation regime
CA3125485A1 (en) 2019-01-11 2020-07-16 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
AU2020256225B9 (en) 2019-04-03 2025-04-10 Regeneron Pharmaceuticals, Inc. Methods and compositions for insertion of antibody coding sequences into a safe harbor locus
AU2020265749A1 (en) 2019-05-01 2022-01-06 Juno Therapeutics, Inc. Cells expressing a chimeric receptor from a modified CD247 locus, related polynucleotides and methods
SG11202111360YA (en) 2019-05-01 2021-11-29 Juno Therapeutics Inc Cells expressing a recombinant receptor from a modified tgfbr2 locus, related polynucleotides and methods
WO2020243261A1 (en) 2019-05-28 2020-12-03 Selecta Biosciences, Inc. Methods and compositions for attenuated anti-viral transfer vector immune response
SG11202111943UA (en) 2019-07-02 2021-11-29 Hutchinson Fred Cancer Res Recombinant ad35 vectors and related gene therapy improvements
CN114728181B (zh) * 2019-07-17 2024-10-01 卡斯西部储备大学 用于治疗hiv感染的长期存活的t细胞
WO2021072115A1 (en) 2019-10-08 2021-04-15 Regents Of The University Of Minnesota Crispr-mediated human genome editing with vectors
AU2020379046B2 (en) 2019-11-08 2025-03-13 Regeneron Pharmaceuticals, Inc. CRISPR and AAV strategies for X-linked juvenile retinoschisis therapy
WO2021108363A1 (en) 2019-11-25 2021-06-03 Regeneron Pharmaceuticals, Inc. Crispr/cas-mediated upregulation of humanized ttr allele
WO2021195079A1 (en) 2020-03-23 2021-09-30 Regeneron Pharmaceuticals, Inc. Non-human animals comprising a humanized ttr locus comprising a v30m mutation and methods of use
JP2023526278A (ja) 2020-05-13 2023-06-21 ジュノー セラピューティクス インコーポレイテッド 組み換え受容体を発現しているドナーバッチ細胞を産生するための方法
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
WO2021234388A1 (en) 2020-05-21 2021-11-25 Oxford Genetics Limited Hdr enhancers
GB202007577D0 (en) 2020-05-21 2020-07-08 Oxford Genetics Ltd Hdr enhancers
JP2023526670A (ja) 2020-05-22 2023-06-22 インシトロ インコーポレイテッド 機械学習モデルを使用した疾患の転帰の予測
EP4171616A1 (de) 2020-06-26 2023-05-03 Juno Therapeutics GmbH Manipulierte t-zellen zur bedingten expression eines rekombinanten rezeptors, zugehörige polynukleotide und verfahren
GB202010075D0 (en) 2020-07-01 2020-08-12 Imp College Innovations Ltd Therapeutic nucleic acids, peptides and uses
US20230374483A1 (en) 2020-07-08 2023-11-23 Regents Of The University Of Minnesota Modified hexosaminidase and uses thereof
DK4182297T3 (da) 2020-07-16 2025-12-01 Acuitas Therapeutics Inc Kationiske lipider til brug i lipid-nanopartikler
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
US12305184B2 (en) 2021-09-03 2025-05-20 North Carolina State University Compositions and methods for conferring resistance to geminivirus
WO2023064367A1 (en) 2021-10-12 2023-04-20 Selecta Biosciences, Inc. Methods and compositions for attenuating anti-viral transfer vector igm responses
WO2023077012A1 (en) 2021-10-27 2023-05-04 Regeneron Pharmaceuticals, Inc. Compositions and methods for expressing factor ix for hemophilia b therapy
CA3237482A1 (en) 2021-11-03 2023-05-11 The J. David Gladstone Institutes, A Testamentary Trust Established Under The Will Of J. David Gladstone Precise genome editing using retrons
WO2023081900A1 (en) 2021-11-08 2023-05-11 Juno Therapeutics, Inc. Engineered t cells expressing a recombinant t cell receptor (tcr) and related systems and methods
KR20240117571A (ko) 2021-12-08 2024-08-01 리제너론 파마슈티칼스 인코포레이티드 돌연변이 마이오실린 질환 모델 및 이의 용도
WO2023114943A2 (en) 2021-12-16 2023-06-22 Acuitas Therapeutics, Inc. Lipids for use in lipid nanoparticle formulations
WO2023122805A1 (en) 2021-12-20 2023-06-29 Vestaron Corporation Sorbitol driven selection pressure method
WO2023141602A2 (en) 2022-01-21 2023-07-27 Renagade Therapeutics Management Inc. Engineered retrons and methods of use
JP2025508677A (ja) 2022-02-02 2025-04-10 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 ポンペ病の処置のための抗ＴｆＲ：ＧＡＡ及び抗ＣＤ６３：ＧＡＡの挿入
US20250194571A1 (en) 2022-02-07 2025-06-19 Regeneron Pharmaceuticals, Inc. Compositions and methods for defining optimal treatment timeframes in lysosomal disease
WO2023150393A2 (en) 2022-02-07 2023-08-10 Ensoma, Inc. Inhibitor-resistant mgmt modifications and modification of mgmt-encoding nucleic acids
JP2025506095A (ja) 2022-02-11 2025-03-07 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 ４ｒタウ標的化剤をスクリーニングするための組成物及び方法
WO2023172624A1 (en) 2022-03-09 2023-09-14 Selecta Biosciences, Inc. Immunosuppressants in combination with anti-igm agents and related dosing
WO2023220043A1 (en) 2022-05-09 2023-11-16 Synteny Therapeutics, Inc. Erythroparvovirus with a modified genome for gene therapy
EP4522201A1 (de) 2022-05-09 2025-03-19 Synteny Therapeutics, Inc. Erythroparvoviruszusammensetzungen und verfahren zur gentherapie
WO2023220040A1 (en) 2022-05-09 2023-11-16 Synteny Therapeutics, Inc. Erythroparvovirus with a modified capsid for gene therapy
AU2023314808A1 (en) 2022-07-29 2025-03-20 Regeneron Pharmaceuticals, Inc. Compositions and methods for transferrin receptor (tfr)-mediated delivery to the brain and muscle
WO2024044723A1 (en) 2022-08-25 2024-02-29 Renagade Therapeutics Management Inc. Engineered retrons and methods of use
WO2024073606A1 (en) 2022-09-28 2024-04-04 Regeneron Pharmaceuticals, Inc. Antibody resistant modified receptors to enhance cell-based therapies
EP4612184A1 (de) 2022-11-04 2025-09-10 Regeneron Pharmaceuticals, Inc. An die calcium-spannungsabhängige kanalhilfsuntereinheit gamma 1 (cacng1) bindende proteine und cacng1-vermittelte abgabe an skelettmuskeln
EP4615960A1 (de) 2022-11-09 2025-09-17 C3S2 GmbH Verfahren zur herstellung von manipulierten immunzellen
WO2024107765A2 (en) 2022-11-14 2024-05-23 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
WO2024197242A1 (en) 2023-03-23 2024-09-26 Carbon Biosciences, Inc. Protoparvovirus compositions comprising a protoparvovirus variant vp1 capsid polypeptide and related methods
EP4683931A1 (de) 2023-03-23 2026-01-28 Carbon Biosciences, Inc. Parvoviruszusammensetzungen und zugehörige verfahren zur gentherapie
GB202311501D0 (en) 2023-07-26 2023-09-06 Imperial College Innovations Ltd Zinc finger peptides, encoded nucleic acids, methods and uses
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
AR133384A1 (es) 2023-07-28 2025-09-24 Regeneron Pharma Anti-tfr:esfingomielinasa ácida para el tratamiento de la deficiencia de esfingomielinasa ácida
WO2025049524A1 (en) 2023-08-28 2025-03-06 Regeneron Pharmaceuticals, Inc. Cxcr4 antibody-resistant modified receptors
WO2025049959A2 (en) 2023-09-01 2025-03-06 Renagade Therapeutics Management Inc. Gene editing systems, compositions, and methods for treatment of vexas syndrome
WO2025128981A1 (en) 2023-12-14 2025-06-19 Sohm, Inc. Compositions and methods for genome editing
WO2025132815A1 (en) 2023-12-20 2025-06-26 Novozymes A/S Novel cas nucleases and polynucleotides encoding the same
WO2025174765A1 (en) 2024-02-12 2025-08-21 Renagade Therapeutics Management Inc. Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents
US20250276092A1 (en) 2024-03-01 2025-09-04 Regeneron Pharmaceuticals, Inc. Methods and compositions for re-dosing aav using anti-cd40 antagonistic antibody to suppress host anti-aav antibody response
WO2025235851A1 (en) 2024-05-10 2025-11-13 Juno Therapeutics, Inc. Genetically engineered t cells expressing a cd19 chimeric antigen receptor (car) and uses thereof for allogeneic cell therapy
US20250388890A1 (en) 2024-06-20 2025-12-25 Regeneron Pharmaceuticals, Inc. ASS1 Gene Insertion For The Treatment Of Citrullinemia Type I

Family Cites Families (50)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5422251A (en) * 1986-11-26 1995-06-06 Princeton University Triple-stranded nucleic acids
US5176996A (en) 1988-12-20 1993-01-05 Baylor College Of Medicine Method for making synthetic oligonucleotides which bind specifically to target sites on duplex DNA molecules, by forming a colinear triplex, the synthetic oligonucleotides and methods of use
US5420032A (en) 1991-12-23 1995-05-30 Universitge Laval Homing endonuclease which originates from chlamydomonas eugametos and recognizes and cleaves a 15, 17 or 19 degenerate double stranded nucleotide sequence
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
US5792632A (en) 1992-05-05 1998-08-11 Institut Pasteur Nucleotide sequence encoding the enzyme I-SceI and the uses thereof
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
US5585245A (en) 1994-04-22 1996-12-17 California Institute Of Technology Ubiquitin-based split protein sensor
DE69535829D1 (de) 1994-08-20 2008-10-16 Gendaq Ltd Verbesserung in bezug auf bindungsproteine bei der 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
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
US6342345B1 (en) * 1997-04-02 2002-01-29 The Board Of Trustees Of The Leland Stanford Junior University Detection of molecular interactions by reporter subunit complementation
GB9710807D0 (en) 1997-05-23 1997-07-23 Medical Res Council Nucleic acid binding proteins
GB9710809D0 (en) 1997-05-23 1997-07-23 Medical Res Council Nucleic acid binding proteins
AU752105B2 (en) * 1997-12-05 2002-09-05 Europaisches Laboratorium Fur Molekularbiologie (Embl) Novel dna cloning method
US6410248B1 (en) 1998-01-30 2002-06-25 Massachusetts Institute Of Technology General strategy for selecting high-affinity zinc finger proteins for diverse DNA target sites
US6140081A (en) * 1998-10-16 2000-10-31 The Scripps Research Institute Zinc finger binding domains for GNN
US6534261B1 (en) 1999-01-12 2003-03-18 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
US6453242B1 (en) 1999-01-12 2002-09-17 Sangamo Biosciences, Inc. Selection of sites for targeting by zinc finger proteins and methods of designing zinc finger proteins to bind to preselected sites
US6599692B1 (en) 1999-09-14 2003-07-29 Sangamo Bioscience, Inc. Functional genomics using zinc finger proteins
US7013219B2 (en) 1999-01-12 2006-03-14 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
US20030104526A1 (en) * 1999-03-24 2003-06-05 Qiang Liu Position dependent recognition of GNN nucleotide triplets by zinc fingers
US7030215B2 (en) * 1999-03-24 2006-04-18 Sangamo Biosciences, Inc. Position dependent recognition of GNN nucleotide triplets by zinc fingers
CA2394850C (en) 1999-12-06 2012-02-07 Sangamo Biosciences, Inc. Methods of using randomized libraries of zinc finger proteins for the identification of gene function
EP1254369B1 (de) 2000-02-08 2010-10-06 Sangamo BioSciences, Inc. Zellen zur entdeckung von medikamenten
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
AU2002225187A1 (en) * 2001-01-22 2002-07-30 Sangamo Biosciences, Inc. Zinc finger polypeptides and their use
GB0108491D0 (en) 2001-04-04 2001-05-23 Gendaq Ltd Engineering zinc fingers
WO2003016496A2 (en) * 2001-08-20 2003-02-27 The Scripps Research Institute Zinc finger binding domains for cnn
ATE347593T1 (de) 2002-01-23 2006-12-15 Univ Utah Res Found Zielgerichtete chromosomale mutagenese mit zinkfingernukleasen
EP1504092B2 (de) 2002-03-21 2014-06-25 Sangamo BioSciences, Inc. Verfahren und zusammensetzungen zur verwendung von zinkfinger-endonukleasen zur verbesserung der homologen rekombination
US7947873B2 (en) * 2002-04-17 2011-05-24 Sangamo Biosciences, Inc. Compositions and methods for regulation of plant gamma-tocopherol methyltransferase
WO2004037977A2 (en) 2002-09-05 2004-05-06 California Institute Of Thechnology Use of chimeric nucleases to stimulate gene targeting
WO2004067753A2 (en) * 2003-01-28 2004-08-12 Cellectis Use of meganucleases for inducing homologous recombination ex vivo and in toto in vertebrate somatic tissues and application thereof.
US7888121B2 (en) 2003-08-08 2011-02-15 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
ES2808687T3 (es) * 2003-08-08 2021-03-01 Sangamo Therapeutics Inc Métodos y composiciones para escisión dirigida y recombinación
US8409861B2 (en) 2003-08-08 2013-04-02 Sangamo Biosciences, Inc. Targeted deletion of cellular DNA sequences
US7253273B2 (en) * 2004-04-08 2007-08-07 Sangamo Biosciences, Inc. Treatment of neuropathic pain with zinc finger proteins
EP1913149A4 (de) * 2005-07-26 2009-08-05 Sangamo Biosciences Inc Gezielte integration und expression exogener nukleinsäuresequenzen
CA2626262C (en) * 2005-10-18 2015-09-08 Homme W. Hellinga Rationally-designed meganucleases with altered sequence specificity and dna-binding affinity
EP2019839B1 (de) * 2006-05-25 2011-12-07 Sangamo BioSciences, Inc. Verfahren und zusammensetzungen zur gendeaktivierung

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