JP6954890B2 - ヌクレアーゼ媒介ゲノム遺伝子操作のための送達方法及び組成物 - Google Patents

ヌクレアーゼ媒介ゲノム遺伝子操作のための送達方法及び組成物 Download PDF

Info

Publication number
JP6954890B2
JP6954890B2 JP2018501179A JP2018501179A JP6954890B2 JP 6954890 B2 JP6954890 B2 JP 6954890B2 JP 2018501179 A JP2018501179 A JP 2018501179A JP 2018501179 A JP2018501179 A JP 2018501179A JP 6954890 B2 JP6954890 B2 JP 6954890B2
Authority
JP
Japan
Prior art keywords
cells
cell
gene
nuclease
donor
Prior art date
Legal status (The legal status 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 status listed.)
Active
Application number
JP2018501179A
Other languages
English (en)
Japanese (ja)
Other versions
JP2018525979A5 (https=
JP2018525979A (ja
Inventor
ゲイリー ケー. リー,
ゲイリー ケー. リー,
ブリジット イー. レイリー,
ブリジット イー. レイリー,
マーティン, スーザン ジェイ. セント
マーティン, スーザン ジェイ. セント
トーマス ウェクスラー,
トーマス ウェクスラー,
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Sangamo Therapeutics Inc
Original Assignee
Sangamo Therapeutics 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 Therapeutics Inc filed Critical Sangamo Therapeutics Inc
Publication of JP2018525979A publication Critical patent/JP2018525979A/ja
Publication of JP2018525979A5 publication Critical patent/JP2018525979A5/ja
Priority to JP2020209010A priority Critical patent/JP7348160B2/ja
Application granted granted Critical
Publication of JP6954890B2 publication Critical patent/JP6954890B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Images

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/36Blood coagulation or fibrinolysis factors
    • A61K38/37Factors VIII
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46Hydrolases (3)
    • A61K38/48Hydrolases (3) acting on peptide bonds (3.4)
    • A61K38/482Serine endopeptidases (3.4.21)
    • A61K38/4846Factor VII (3.4.21.21); Factor IX (3.4.21.22); Factor Xa (3.4.21.6); Factor XI (3.4.21.27); Factor XII (3.4.21.38)
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00Cellular immunotherapy
    • A61K40/10Cellular immunotherapy characterised by the cell type used
    • A61K40/11T-cells, e.g. tumour infiltrating lymphocytes [TIL] or regulatory T [Treg] cells; Lymphokine-activated killer [LAK] cells
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00Cellular immunotherapy
    • A61K40/30Cellular immunotherapy characterised by the recombinant expression of specific molecules in the cells of the immune system
    • A61K40/31Chimeric antigen receptors [CAR]
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00Cellular immunotherapy
    • A61K40/40Cellular immunotherapy characterised by antigens that are targeted or presented by cells of the immune system
    • A61K40/41Vertebrate antigens
    • A61K40/42Cancer antigens
    • A61K40/4202Receptors, cell surface antigens or cell surface determinants
    • A61K40/4203Receptors for growth factors
    • A61K40/4204Epidermal growth factor receptors [EGFR]
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00Cellular immunotherapy
    • A61K40/40Cellular immunotherapy characterised by antigens that are targeted or presented by cells of the immune system
    • A61K40/41Vertebrate antigens
    • A61K40/42Cancer antigens
    • A61K40/4202Receptors, cell surface antigens or cell surface determinants
    • A61K40/4203Receptors for growth factors
    • A61K40/4206Fibroblast growth factor receptors [FGFR]
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00Cellular immunotherapy
    • A61K40/40Cellular immunotherapy characterised by antigens that are targeted or presented by cells of the immune system
    • A61K40/41Vertebrate antigens
    • A61K40/42Cancer antigens
    • A61K40/4202Receptors, cell surface antigens or cell surface determinants
    • A61K40/4203Receptors for growth factors
    • A61K40/4207Platelet-derived growth factor receptors [PDGFR]
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00Cellular immunotherapy
    • A61K40/40Cellular immunotherapy characterised by antigens that are targeted or presented by cells of the immune system
    • A61K40/41Vertebrate antigens
    • A61K40/42Cancer antigens
    • A61K40/4202Receptors, cell surface antigens or cell surface determinants
    • A61K40/4203Receptors for growth factors
    • A61K40/4209Hepatocyte growth factor receptor [HGFR] or c-met]
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00Drugs for disorders of the blood or the extracellular fluid
    • A61P7/04Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N5/00Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06Animal cells or tissues; Human cells or tissues
    • C12N5/0602Vertebrate cells
    • C12N5/0634Cells from the blood or the immune system
    • C12N5/0636T lymphocytes
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y304/00Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/21Serine endopeptidases (3.4.21)
    • C12Y304/21022Coagulation factor IXa (3.4.21.22)
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2239/00Indexing codes associated with cellular immunotherapy of group A61K40/00
    • A61K2239/27Indexing codes associated with cellular immunotherapy of group A61K40/00 characterized by targeting or presenting multiple antigens
    • A61K2239/29Multispecific CARs
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2501/00Active agents used in cell culture processes, e.g. differentation
    • C12N2501/50Cell markers; Cell surface determinants
    • C12N2501/515CD3, T-cell receptor complex
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2510/00Genetically modified cells
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Immunology (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Hematology (AREA)
  • Microbiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Molecular Biology (AREA)
  • Virology (AREA)
  • Cell Biology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Diabetes (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2018501179A 2015-07-13 2016-07-13 ヌクレアーゼ媒介ゲノム遺伝子操作のための送達方法及び組成物 Active JP6954890B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2020209010A JP7348160B2 (ja) 2015-07-13 2020-12-17 ヌクレアーゼ媒介ゲノム遺伝子操作のための送達方法及び組成物

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201562191918P 2015-07-13 2015-07-13
US62/191,918 2015-07-13
US201562247469P 2015-10-28 2015-10-28
US62/247,469 2015-10-28
US201662315438P 2016-03-30 2016-03-30
US62/315,438 2016-03-30
PCT/US2016/042024 WO2017011519A1 (en) 2015-07-13 2016-07-13 Delivery methods and compositions for nuclease-mediated genome engineering

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2020209010A Division JP7348160B2 (ja) 2015-07-13 2020-12-17 ヌクレアーゼ媒介ゲノム遺伝子操作のための送達方法及び組成物

Publications (3)

Publication Number Publication Date
JP2018525979A JP2018525979A (ja) 2018-09-13
JP2018525979A5 JP2018525979A5 (https=) 2019-08-22
JP6954890B2 true JP6954890B2 (ja) 2021-10-27

Family

ID=57758305

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2018501179A Active JP6954890B2 (ja) 2015-07-13 2016-07-13 ヌクレアーゼ媒介ゲノム遺伝子操作のための送達方法及び組成物
JP2020209010A Active JP7348160B2 (ja) 2015-07-13 2020-12-17 ヌクレアーゼ媒介ゲノム遺伝子操作のための送達方法及び組成物

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2020209010A Active JP7348160B2 (ja) 2015-07-13 2020-12-17 ヌクレアーゼ媒介ゲノム遺伝子操作のための送達方法及び組成物

Country Status (7)

Country Link
US (2) US10450585B2 (https=)
EP (1) EP3322297B1 (https=)
JP (2) JP6954890B2 (https=)
CN (1) CN108024544B (https=)
AU (1) AU2016291778B2 (https=)
CA (1) CA2991301A1 (https=)
WO (1) WO2017011519A1 (https=)

Families Citing this family (117)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP6261500B2 (ja) 2011-07-22 2018-01-17 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 ヌクレアーゼ切断特異性の評価および改善
US9950282B2 (en) 2012-03-15 2018-04-24 Flodesign Sonics, Inc. Electronic configuration and control for acoustic standing wave generation
US9752113B2 (en) 2012-03-15 2017-09-05 Flodesign Sonics, Inc. Acoustic perfusion devices
US10689609B2 (en) 2012-03-15 2020-06-23 Flodesign Sonics, Inc. Acoustic bioreactor processes
US10704021B2 (en) 2012-03-15 2020-07-07 Flodesign Sonics, Inc. Acoustic perfusion devices
US9458450B2 (en) 2012-03-15 2016-10-04 Flodesign Sonics, Inc. Acoustophoretic separation technology using multi-dimensional standing waves
US9745548B2 (en) 2012-03-15 2017-08-29 Flodesign Sonics, Inc. Acoustic perfusion devices
US10967298B2 (en) 2012-03-15 2021-04-06 Flodesign Sonics, Inc. Driver and control for variable impedence load
US10322949B2 (en) 2012-03-15 2019-06-18 Flodesign Sonics, Inc. Transducer and reflector configurations for an acoustophoretic device
US10737953B2 (en) 2012-04-20 2020-08-11 Flodesign Sonics, Inc. Acoustophoretic method for use in bioreactors
US20150044192A1 (en) 2013-08-09 2015-02-12 President And Fellows Of Harvard College Methods for identifying a target site of a cas9 nuclease
US9359599B2 (en) 2013-08-22 2016-06-07 President And Fellows Of Harvard College Engineered transcription activator-like effector (TALE) domains and uses thereof
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
US9388430B2 (en) 2013-09-06 2016-07-12 President And Fellows Of Harvard College Cas9-recombinase fusion proteins and uses thereof
US9340799B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College MRNA-sensing switchable gRNAs
US9745569B2 (en) 2013-09-13 2017-08-29 Flodesign Sonics, Inc. System for generating high concentration factors for low cell density suspensions
US20150165054A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting caspase-9 point mutations
WO2015105955A1 (en) 2014-01-08 2015-07-16 Flodesign Sonics, Inc. Acoustophoresis device with dual acoustophoretic chamber
US9744483B2 (en) 2014-07-02 2017-08-29 Flodesign Sonics, Inc. Large scale acoustic separation device
WO2016022363A2 (en) 2014-07-30 2016-02-11 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
EP3204496A1 (en) 2014-10-10 2017-08-16 Editas Medicine, Inc. Compositions and methods for promoting homology directed repair
CA2963820A1 (en) 2014-11-07 2016-05-12 Editas Medicine, Inc. Methods for improving crispr/cas-mediated genome-editing
GB201420139D0 (en) 2014-11-12 2014-12-24 Ucl Business Plc Factor IX gene therapy
US11021699B2 (en) 2015-04-29 2021-06-01 FioDesign Sonics, Inc. Separation using angled acoustic waves
US11708572B2 (en) 2015-04-29 2023-07-25 Flodesign Sonics, Inc. Acoustic cell separation techniques and processes
US11377651B2 (en) 2016-10-19 2022-07-05 Flodesign Sonics, Inc. Cell therapy processes utilizing acoustophoresis
EP3322297B1 (en) 2015-07-13 2024-12-04 Sangamo Therapeutics, Inc. Delivery methods and compositions for nuclease-mediated genome engineering
US11459540B2 (en) 2015-07-28 2022-10-04 Flodesign Sonics, Inc. Expanded bed affinity selection
US11474085B2 (en) 2015-07-28 2022-10-18 Flodesign Sonics, Inc. Expanded bed affinity selection
GB2592821B (en) 2015-07-31 2022-01-12 Univ Minnesota Modified cells and methods of therapy
AU2016326711B2 (en) 2015-09-24 2022-11-03 Editas Medicine, Inc. Use of exonucleases to improve CRISPR/Cas-mediated genome editing
CN117070468A (zh) 2015-10-05 2023-11-17 精密生物科学公司 包含经修饰的人T细胞受体α恒定区基因的经遗传修饰的细胞
EP3359660B1 (en) 2015-10-05 2019-12-04 Precision Biosciences, Inc. Engineered meganucleases with recognition sequences found in the human t cell receptor alpha constant region gene
JP7109784B2 (ja) 2015-10-23 2022-08-01 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 遺伝子編集のための進化したCas9蛋白質
CA3002524A1 (en) 2015-10-28 2017-05-04 Sangamo Therapeutics, Inc. Liver-specific constructs, factor viii expression cassettes and methods of use thereof
EP3433363A1 (en) 2016-03-25 2019-01-30 Editas Medicine, Inc. Genome editing systems comprising repair-modulating enzyme molecules and methods of their use
WO2017180694A1 (en) 2016-04-13 2017-10-19 Editas Medicine, Inc. Cas9 fusion molecules gene editing systems, and methods of use thereof
US11377637B2 (en) 2016-04-15 2022-07-05 Memorial Sloan Kettering Cancer Center Transgenic T cell and chimeric antigen receptor T cell compositions and related methods
US11085035B2 (en) 2016-05-03 2021-08-10 Flodesign Sonics, Inc. Therapeutic cell washing, concentration, and separation utilizing acoustophoresis
US11214789B2 (en) 2016-05-03 2022-01-04 Flodesign Sonics, Inc. Concentration and washing of particles with acoustics
KR102547316B1 (ko) 2016-08-03 2023-06-23 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 아데노신 핵염기 편집제 및 그의 용도
US11661590B2 (en) 2016-08-09 2023-05-30 President And Fellows Of Harvard College Programmable CAS9-recombinase fusion proteins and uses thereof
SG10201913948PA (en) * 2016-08-24 2020-03-30 Sangamo Therapeutics Inc Engineered target specific nucleases
US11542509B2 (en) 2016-08-24 2023-01-03 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
LT3504229T (lt) 2016-08-24 2021-12-10 Sangamo Therapeutics, Inc. Genų raiškos reguliavimas, panaudojant rekombinantines nukleazes
CN110139873A (zh) 2016-10-03 2019-08-16 朱诺治疗学股份有限公司 Hpv特异性结合分子
EP3526320A1 (en) 2016-10-14 2019-08-21 President and Fellows of Harvard College Aav delivery of nucleobase editors
JP7181862B2 (ja) 2016-10-18 2022-12-01 リージェンツ オブ ザ ユニバーシティ オブ ミネソタ 腫瘍浸潤リンパ球および治療の方法
CA3041517A1 (en) 2016-10-19 2018-04-26 Flodesign Sonics, Inc. Affinity cell extraction by acoustics
US20200087681A1 (en) * 2016-12-21 2020-03-19 Ucl Business Plc Therapeutic cells
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
WO2018140573A1 (en) * 2017-01-26 2018-08-02 Sangamo Therapeutics, Inc. B-cell engineering
JP2020510038A (ja) 2017-03-09 2020-04-02 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 がんワクチン
EP3592853A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Suppression of pain by gene editing
JP2020510439A (ja) 2017-03-10 2020-04-09 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 シトシンからグアニンへの塩基編集因子
CA3057192A1 (en) 2017-03-23 2018-09-27 President And Fellows Of Harvard College Nucleobase editors comprising nucleic acid programmable dna binding proteins
WO2018209320A1 (en) 2017-05-12 2018-11-15 President And Fellows Of Harvard College Aptazyme-embedded guide rnas for use with crispr-cas9 in genome editing and transcriptional activation
CA3067382A1 (en) 2017-06-15 2018-12-20 The Regents Of The University Of California Targeted non-viral dna insertions
BR112019026622A2 (pt) * 2017-06-16 2020-06-30 Sangamo Therapeutics, Inc. disrupção direcionada de receptores de célula t e/ou hla
CA3068465A1 (en) 2017-06-30 2019-01-03 Precision Biosciences, Inc. Genetically-modified t cells comprising a modified intron in the t cell receptor alpha gene
WO2019006418A2 (en) 2017-06-30 2019-01-03 Intima Bioscience, Inc. ADENO-ASSOCIATED VIRAL VECTORS FOR GENE THERAPY
WO2019006330A1 (en) * 2017-06-30 2019-01-03 Indiana University Research And Technology Corporation COMPOSITIONS AND METHODS FOR DETECTING REACTIVITY TO ALS
US11866726B2 (en) 2017-07-14 2024-01-09 Editas Medicine, Inc. Systems and methods for targeted integration and genome editing and detection thereof using integrated priming sites
WO2019023680A1 (en) 2017-07-28 2019-01-31 President And Fellows Of Harvard College METHODS AND COMPOSITIONS FOR EVOLUTION OF BASIC EDITORS USING PHAGE-ASSISTED CONTINUOUS EVOLUTION (PACE)
EP3676376B1 (en) 2017-08-30 2025-01-15 President and Fellows of Harvard College High efficiency base editors comprising gam
TW201920661A (zh) * 2017-09-18 2019-06-01 大陸商博雅輯因(北京)生物科技有限公司 一種基因編輯t細胞及其用途
TWI835747B (zh) 2017-09-22 2024-03-21 賓州大學委員會 用於治療黏多醣病 ii 型之基因治療
BR112020006643A2 (pt) 2017-10-03 2020-09-24 Juno Therapeutics Inc moléculas de ligação específica ao hpv
KR20250107288A (ko) 2017-10-16 2025-07-11 더 브로드 인스티튜트, 인코퍼레이티드 아데노신 염기 편집제의 용도
KR102503130B1 (ko) 2017-10-27 2023-02-24 더 리전트 오브 더 유니버시티 오브 캘리포니아 내인성 t 세포 수용체의 표적화된 대체
WO2019118516A1 (en) * 2017-12-11 2019-06-20 Editas Medicine, Inc. Cpf1-related methods and compositions for gene editing
WO2019118921A1 (en) 2017-12-14 2019-06-20 Flodesign Sonics, Inc. Acoustic transducer drive and controller
WO2019118949A1 (en) 2017-12-15 2019-06-20 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
CA3094468A1 (en) 2018-04-05 2019-10-10 Juno Therapeutics, Inc. Methods of producing cells expressing a recombinant receptor and related compositions
AU2019249209A1 (en) 2018-04-05 2020-10-15 Juno Therapeutics, Inc. T cell receptors and engineered cells expressing same
KR20210020873A (ko) 2018-04-05 2021-02-24 주노 쎄러퓨티크스 인코퍼레이티드 재조합 수용체를 발현하는 τ 세포, 관련 폴리뉴클레오티드 및 방법
CA3095795A1 (en) 2018-04-12 2019-10-17 Precision Biosciences, Inc. Optimized engineered nucleases having specificity for the human t cell receptor alpha constant region gene
EP3797160A1 (en) 2018-05-23 2021-03-31 The Broad Institute Inc. Base editors and uses thereof
KR20240014102A (ko) 2018-06-29 2024-01-31 우한 뉴로프스 바이오테크놀로지 리미티드 컴퍼니 레버 유전성 시신경병증의 치료를 위한 조성물 및 방법
US12522807B2 (en) 2018-07-09 2026-01-13 The Broad Institute, Inc. RNA programmable epigenetic RNA modifiers and uses thereof
EP3820509A1 (en) 2018-07-09 2021-05-19 Flagship Pioneering Innovations V, Inc. Fusosome compositions and uses thereof
WO2020028830A1 (en) 2018-08-03 2020-02-06 Sangamo Therapeutics, Inc. Improved clinical parameters by expression of factor viii
US20200063160A1 (en) * 2018-08-07 2020-02-27 Sangamo Therapeutics, Inc. Method for the treatment of mucopolysaccharidosis type ii
CN108949763A (zh) * 2018-08-15 2018-12-07 吉林大学 能有效抑制猪瘟病毒感染的精确突变LamR基因及应用
CA3109432A1 (en) 2018-08-20 2020-02-27 Wuhan Neurophth Biotechnology Limited Company Compositions and methods for treating leber's hereditary optic neuropathy
WO2020038352A1 (en) * 2018-08-20 2020-02-27 Wuhan Neurophth Biological Technology Limited Company Compositions and methods for treating leber's hereditary optic neuropathy
US10842885B2 (en) 2018-08-20 2020-11-24 Ucl Business Ltd Factor IX encoding nucleotides
GB201813528D0 (en) 2018-08-20 2018-10-03 Ucl Business Plc Factor IX encoding nucleotides
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
WO2020092453A1 (en) 2018-10-29 2020-05-07 The Broad Institute, Inc. Nucleobase editors comprising geocas9 and uses thereof
US12496274B2 (en) 2018-11-14 2025-12-16 Flagship Pioneering Innovations V, Inc. Compositions and methods for compartment-specific cargo delivery
WO2020102503A2 (en) * 2018-11-14 2020-05-22 Flagship Pioneering Innovations V, Inc. Fusosome compositions for t cell delivery
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
EP3826664A4 (en) 2019-02-06 2022-10-26 Sangamo Therapeutics, Inc. Method for the treatment of mucopolysaccharidosis type i
JP7657726B2 (ja) 2019-03-19 2025-04-07 ザ ブロード インスティテュート,インコーポレーテッド 編集ヌクレオチド配列を編集するための方法および組成物
US12473543B2 (en) 2019-04-17 2025-11-18 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
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
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
US11622547B2 (en) 2019-06-07 2023-04-11 Regeneran Pharmaceuticals, Inc. Genetically modified mouse that expresses human albumin
CN114502587A (zh) * 2019-07-30 2022-05-13 普瑞文森生物有限公司 通过非耗竭性b细胞抑制剂降低免疫原性的方法和组合物
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
CN113025633B (zh) 2019-12-09 2024-08-27 武汉纽福斯生物科技有限公司 编码人nadh脱氢酶亚单位1蛋白的核酸及其应用
DE112021002672T5 (de) 2020-05-08 2023-04-13 President And Fellows Of Harvard College Vefahren und zusammensetzungen zum gleichzeitigen editieren beider stränge einer doppelsträngigen nukleotid-zielsequenz
WO2021231661A2 (en) 2020-05-13 2021-11-18 Juno Therapeutics, Inc. Process for producing donor-batched cells expressing a recombinant receptor
EP4171616A1 (en) 2020-06-26 2023-05-03 Juno Therapeutics GmbH Engineered t cells conditionally expressing a recombinant receptor, related polynucleotides and methods
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
AU2022366987A1 (en) 2021-10-14 2024-05-16 Arsenal Biosciences, Inc. Immune cells having co-expressed shrnas and logic gate systems
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
WO2023122036A1 (en) * 2021-12-20 2023-06-29 Sobol Robert E Methods and compositions for improved molecular therapies of multigenic diseases
CN119546757A (zh) 2022-05-25 2025-02-28 宏基因组学公司 肝酶表达的补充
EP4615960A1 (en) 2022-11-09 2025-09-17 C3S2 GmbH Methods for manufacturing engineered immune cells
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
WO2024229281A1 (en) * 2023-05-02 2024-11-07 City Of Hope Adeno-associated virus compositions and methods of use thereof
WO2025096638A2 (en) 2023-10-30 2025-05-08 Turnstone Biologics Corp. Genetically modified tumor infilitrating lymphocytes and methods of producing and using the same
WO2025117858A1 (en) * 2023-11-28 2025-06-05 Metagenomi, Inc. Supplementation of liver enzyme expression
WO2025212519A1 (en) 2024-04-01 2025-10-09 Moonlight Bio, Inc. Dll3 binding proteins and uses thereof
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

Family Cites Families (120)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US255250A (en) 1882-03-21 Whistling toy
US4217344A (en) 1976-06-23 1980-08-12 L'oreal Compositions containing aqueous dispersions of lipid spheres
US4235871A (en) 1978-02-24 1980-11-25 Papahadjopoulos Demetrios P Method of encapsulating biologically active materials in lipid vesicles
US4186183A (en) 1978-03-29 1980-01-29 The United States Of America As Represented By The Secretary Of The Army Liposome carriers in chemotherapy of leishmaniasis
US4261975A (en) 1979-09-19 1981-04-14 Merck & Co., Inc. Viral liposome particle
US4485054A (en) 1982-10-04 1984-11-27 Lipoderm Pharmaceuticals Limited Method of encapsulating biologically active materials in multilamellar lipid vesicles (MLV)
US4501728A (en) 1983-01-06 1985-02-26 Technology Unlimited, Inc. Masking of liposomes from RES recognition
US4897355A (en) 1985-01-07 1990-01-30 Syntex (U.S.A.) Inc. N[ω,(ω-1)-dialkyloxy]- and N-[ω,(ω-1)-dialkenyloxy]-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US4946787A (en) 1985-01-07 1990-08-07 Syntex (U.S.A.) Inc. N-(ω,(ω-1)-dialkyloxy)- and N-(ω,(ω-1)-dialkenyloxy)-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US5049386A (en) 1985-01-07 1991-09-17 Syntex (U.S.A.) Inc. N-ω,(ω-1)-dialkyloxy)- and N-(ω,(ω-1)-dialkenyloxy)Alk-1-YL-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US4797368A (en) 1985-03-15 1989-01-10 The United States Of America As Represented By The Department Of Health And Human Services Adeno-associated virus as eukaryotic expression vector
US4774085A (en) 1985-07-09 1988-09-27 501 Board of Regents, Univ. of Texas Pharmaceutical administration systems containing a mixture of immunomodulators
US5422251A (en) 1986-11-26 1995-06-06 Princeton University Triple-stranded nucleic acids
US4837028A (en) 1986-12-24 1989-06-06 Liposome Technology, Inc. Liposomes with enhanced circulation time
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
US5264618A (en) 1990-04-19 1993-11-23 Vical, Inc. Cationic lipids for intracellular delivery of biologically active molecules
WO1991017424A1 (en) 1990-05-03 1991-11-14 Vical, Inc. Intracellular delivery of biologically active substances by means of self-assembling lipid complexes
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
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
US5487994A (en) 1992-04-03 1996-01-30 The Johns Hopkins University Insertion and deletion mutants of FokI restriction endonuclease
US5436150A (en) 1992-04-03 1995-07-25 The Johns Hopkins University Functional domains in flavobacterium okeanokoities (foki) restriction endonuclease
US5356802A (en) 1992-04-03 1994-10-18 The Johns Hopkins University Functional domains in flavobacterium okeanokoites (FokI) restriction endonuclease
US5792632A (en) 1992-05-05 1998-08-11 Institut Pasteur Nucleotide sequence encoding the enzyme I-SceI and the uses thereof
US5587308A (en) 1992-06-02 1996-12-24 The United States Of America As Represented By The Department Of Health & Human Services Modified adeno-associated virus vector capable of expression from a novel promoter
US6242568B1 (en) 1994-01-18 2001-06-05 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
DE69534629D1 (de) 1994-01-18 2005-12-29 Scripps Research Inst Derivate von zinkfingerproteinen und methoden
JP4285766B2 (ja) 1994-03-23 2009-06-24 オハイオ ユニバーシティ 緻密にした核酸および細胞へのデリバリ
US5585245A (en) 1994-04-22 1996-12-17 California Institute Of Technology Ubiquitin-based split protein sensor
GB9824544D0 (en) 1998-11-09 1999-01-06 Medical Res Council Screening system
ATE524545T1 (de) 1994-08-20 2011-09-15 Gendaq Ltd Verbesserungen bei oder im zusammenhang mit bindeproteinen zur erkennung von 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
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
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
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
US6140815A (en) 1998-06-17 2000-10-31 Dover Instrument Corporation High stability spin stand platform
US6140081A (en) 1998-10-16 2000-10-31 The Scripps Research Institute Zinc finger binding domains for GNN
AU770899B2 (en) * 1998-11-10 2004-03-04 Licentia Ltd Platelet-derived growth factor D, DNA coding therefor, and uses thereof
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
US7070934B2 (en) 1999-01-12 2006-07-04 Sangamo Biosciences, Inc. Ligand-controlled regulation of endogenous gene expression
US7013219B2 (en) 1999-01-12 2006-03-14 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
US6534261B1 (en) 1999-01-12 2003-03-18 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
US6599692B1 (en) 1999-09-14 2003-07-29 Sangamo Bioscience, Inc. Functional genomics using zinc finger proteins
US6794136B1 (en) 2000-11-20 2004-09-21 Sangamo Biosciences, Inc. Iterative optimization in the design of binding proteins
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
KR20020086508A (ko) 2000-02-08 2002-11-18 상가모 바이오사이언스 인코포레이티드 약물 발견용 세포
US20020061512A1 (en) 2000-02-18 2002-05-23 Kim Jin-Soo Zinc finger domains and methods of identifying same
EP1295463A4 (en) 2000-05-16 2005-12-14 Comsonics Inc DEVICE AND METHOD FOR DETERMINING THE LOCATION OF SIGNAL ENTRY
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
US20050267061A1 (en) 2004-04-08 2005-12-01 Sangamo Biosciences, Inc. Methods and compositions for treating neuropathic and neurodegenerative conditions
US7067317B2 (en) 2000-12-07 2006-06-27 Sangamo Biosciences, Inc. Regulation of angiogenesis with zinc finger proteins
GB0108491D0 (en) 2001-04-04 2001-05-23 Gendaq Ltd Engineering zinc fingers
US20030003583A1 (en) * 2001-06-08 2003-01-02 Raphael Hirsch Regulation of transgene expression following AAV transduction
US20040224385A1 (en) 2001-08-20 2004-11-11 Barbas Carlos F Zinc finger binding domains for cnn
US7262054B2 (en) 2002-01-22 2007-08-28 Sangamo Biosciences, Inc. Zinc finger proteins for DNA binding and gene regulation in plants
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
US7361635B2 (en) 2002-08-29 2008-04-22 Sangamo Biosciences, Inc. Simultaneous modulation of multiple genes
EP2806025B1 (en) 2002-09-05 2019-04-03 California Institute of Technology Use of zinc finger nucleases to stimulate gene targeting
GB0312086D0 (en) * 2003-05-27 2003-07-02 Novartis Ag Organic compounds
US7888121B2 (en) 2003-08-08 2011-02-15 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
US8409861B2 (en) 2003-08-08 2013-04-02 Sangamo Biosciences, Inc. Targeted deletion of cellular DNA sequences
US7972854B2 (en) 2004-02-05 2011-07-05 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
JP2008506359A (ja) 2004-04-08 2008-03-06 サンガモ バイオサイエンシズ インコーポレイテッド ジンクフィンガータンパク質による神経因性疼痛の処置
BRPI0508762A (pt) * 2004-04-16 2007-08-14 Genentech Inc método de aumento do esgotamento de células b em mamìferos, método de aumento da eficácia do esgotamento de células b, método de tratamento de malignidade ou neoplasma de células b, método de alìvio de disfunção autoimunológica regulada por células b, método de esgotamento das células b e composição
KR20070060115A (ko) 2004-09-16 2007-06-12 상가모 바이오사이언스 인코포레이티드 단백질 생산을 위한 조성물 및 방법
EP1877583A2 (en) 2005-05-05 2008-01-16 Arizona Board of Regents on behalf of the Unversity of Arizona Sequence enabled reassembly (seer) - a novel method for visualizing specific dna sequences
AU2006272634B2 (en) 2005-07-26 2013-01-24 Sangamo Therapeutics, Inc. Targeted integration and expression of exogenous nucleic acid sequences
CA2891996A1 (en) 2005-10-18 2007-04-26 Duke University Rationally-designed meganucleases with altered sequence specificity and dna-binding affinity
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
ES2465996T3 (es) 2006-05-25 2014-06-09 Sangamo Biosciences, Inc. Métodos y composiciones para la inactivación genética
WO2008133938A2 (en) 2007-04-26 2008-11-06 Sangamo Biosciences, Inc. Targeted integration into the ppp1r12c locus
US8790345B2 (en) 2007-08-21 2014-07-29 Zimmer, Inc. Titanium alloy with oxidized zirconium for a prosthetic implant
US8563314B2 (en) 2007-09-27 2013-10-22 Sangamo Biosciences, Inc. Methods and compositions for modulating PD1
EP2281050B1 (en) 2008-04-14 2014-04-02 Sangamo BioSciences, Inc. Linear donor constructs for targeted integration
SG191561A1 (en) 2008-08-22 2013-07-31 Sangamo Biosciences Inc Methods and compositions for targeted single-stranded cleavage and targeted integration
KR101297192B1 (ko) 2008-11-10 2013-08-19 삼성전자주식회사 화상형성장치, 칩, 및, 칩 패키지
ES2498340T3 (es) 2008-11-17 2014-09-24 Koninklijke Nederlandse Akademie Van Wetenschappen Métodos para identificar compuestos de modulación de la linfangiogénesis, medios para ello, compuestos y usos de estos
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
EP2206723A1 (en) 2009-01-12 2010-07-14 Bonas, Ulla Modular DNA-binding domains
US8956828B2 (en) 2009-11-10 2015-02-17 Sangamo Biosciences, Inc. Targeted disruption of T cell receptor genes using engineered zinc finger protein nucleases
EP3594333B1 (en) 2010-02-08 2023-11-01 Sangamo Therapeutics, Inc. Engineered cleavage half-domains
EP2534163B1 (en) 2010-02-09 2015-11-04 Sangamo BioSciences, Inc. Targeted genomic modification with partially single-stranded donor molecules
JP2013523776A (ja) * 2010-04-02 2013-06-17 セレジーン インコーポレイテッド パーキンソン病および脳のドーパミン作動性ニューロンのその他の障害を処置する方法
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
US8808684B2 (en) * 2010-09-10 2014-08-19 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Epidermal growth factor receptor (EGFR) and methods of use in adenoviral-associated virus type 6 (AAV6) transduction
US9405700B2 (en) 2010-11-04 2016-08-02 Sonics, Inc. Methods and apparatus for virtualization in an integrated circuit
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
AU2012340213B2 (en) 2011-11-16 2017-12-07 Sangamo Therapeutics, Inc. Modified DNA-binding proteins and uses thereof
CN102633883B (zh) * 2012-02-24 2014-06-25 上海白泽生物科技有限公司 一种能与egfr、her2、vegf高效结合的融合蛋白、其编码序列及用途
AU2013259647B2 (en) 2012-05-07 2018-11-08 Corteva Agriscience Llc Methods and compositions for nuclease-mediated targeted integration of transgenes
KR102437522B1 (ko) * 2012-05-25 2022-08-26 셀렉티스 면역요법을 위한 동종이형 및 면역억제제 저항성인 t 세포의 조작 방법
US10648001B2 (en) 2012-07-11 2020-05-12 Sangamo Therapeutics, Inc. Method of treating mucopolysaccharidosis type I or II
EP2906684B8 (en) * 2012-10-10 2020-09-02 Sangamo Therapeutics, Inc. T cell modifying compounds and uses thereof
WO2014089212A1 (en) 2012-12-05 2014-06-12 Sangamo Biosciences, Inc. Methods and compositions for regulation of metabolic disorders
EP2928303A4 (en) 2012-12-07 2016-07-13 Haplomics Inc FACTOR VIII MUTATION REPAIR AND TOLERANCE INDUCTION
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
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
US9999206B2 (en) * 2013-07-25 2018-06-19 Utah State University Model and method for a transgenic bovidae expressing cardiac fibrosis and associated pathology
DK3080274T3 (da) 2013-12-09 2020-08-31 Sangamo Therapeutics Inc Fremgangsmåder og sammensætninger til genom-manipulation
AU2015218576B2 (en) 2014-02-24 2020-02-27 Sangamo Therapeutics, Inc. Methods and compositions for nuclease-mediated targeted integration
US20170049887A1 (en) * 2014-04-25 2017-02-23 University Of Florida Research Foundation, Inc. Methods of permitting a subject to receive multiple doses of recombinant adeno-associated virus
RU2691102C2 (ru) 2014-05-08 2019-06-11 Сангамо Байосайенсиз, Инк. Способы и композиции для лечения болезни хантингтона
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
EP3322297B1 (en) 2015-07-13 2024-12-04 Sangamo Therapeutics, Inc. Delivery methods and compositions for nuclease-mediated genome engineering
CN117070468A (zh) * 2015-10-05 2023-11-17 精密生物科学公司 包含经修饰的人T细胞受体α恒定区基因的经遗传修饰的细胞
CA3002524A1 (en) 2015-10-28 2017-05-04 Sangamo Therapeutics, Inc. Liver-specific constructs, factor viii expression cassettes and methods of use thereof

Also Published As

Publication number Publication date
EP3322297A1 (en) 2018-05-23
AU2016291778B2 (en) 2021-05-06
EP3322297A4 (en) 2019-04-17
AU2016291778A1 (en) 2018-01-25
CN108024544A (zh) 2018-05-11
US10450585B2 (en) 2019-10-22
JP2021045166A (ja) 2021-03-25
EP3322297B1 (en) 2024-12-04
US20200102577A1 (en) 2020-04-02
EP3322297C0 (en) 2024-12-04
JP2018525979A (ja) 2018-09-13
CA2991301A1 (en) 2017-01-19
JP7348160B2 (ja) 2023-09-20
WO2017011519A1 (en) 2017-01-19
CN108024544B (zh) 2022-04-29
US20170016027A1 (en) 2017-01-19

Similar Documents

Publication Publication Date Title
JP7348160B2 (ja) ヌクレアーゼ媒介ゲノム遺伝子操作のための送達方法及び組成物
JP6875362B2 (ja) ヌクレアーゼ媒介ゲノム遺伝子操作のための送達方法および組成物
US20230203540A1 (en) Methods and compositions for nuclease-mediated targeted integration of transgenes into mammalian liver cells
CN105208866B (zh) 使用工程化锌指蛋白核酸酶靶向断裂t细胞受体基因
JP6722176B2 (ja) 造血幹細胞におけるヌクレアーゼ媒介性ゲノム工学および補正のための方法および組成物
US20150174169A1 (en) Delivery methods and compositions for nuclease-mediated genome engineering in hematopoietic stem cells
JP2022105621A (ja) 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
HK1260200A1 (en) Targeted disruption of the mhc cell receptor

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20190712

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20190712

A977 Report on retrieval

Free format text: JAPANESE INTERMEDIATE CODE: A971007

Effective date: 20200424

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20200617

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20200916

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20201116

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20201217

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20210315

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20210708

C60 Trial request (containing other claim documents, opposition documents)

Free format text: JAPANESE INTERMEDIATE CODE: C60

Effective date: 20210708

A911 Transfer to examiner for re-examination before appeal (zenchi)

Free format text: JAPANESE INTERMEDIATE CODE: A911

Effective date: 20210715

C21 Notice of transfer of a case for reconsideration by examiners before appeal proceedings

Free format text: JAPANESE INTERMEDIATE CODE: C21

Effective date: 20210716

TRDD Decision of grant or rejection written
A01 Written decision to grant a patent or to grant a registration (utility model)

Free format text: JAPANESE INTERMEDIATE CODE: A01

Effective date: 20210902

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20210930

R150 Certificate of patent or registration of utility model

Ref document number: 6954890

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250