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
JP2018525979A (ja
JP2018525979A5 (cg-RX-API-DMAC7.html
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)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Molecular Biology (AREA)
  • Virology (AREA)
  • Cell Biology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Diabetes (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (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 (cg-RX-API-DMAC7.html) 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 (cg-RX-API-DMAC7.html)
EP (1) EP3322297B1 (cg-RX-API-DMAC7.html)
JP (2) JP6954890B2 (cg-RX-API-DMAC7.html)
CN (1) CN108024544B (cg-RX-API-DMAC7.html)
AU (1) AU2016291778B2 (cg-RX-API-DMAC7.html)
CA (1) CA2991301A1 (cg-RX-API-DMAC7.html)
WO (1) WO2017011519A1 (cg-RX-API-DMAC7.html)

Families Citing this family (115)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2734621B1 (en) 2011-07-22 2019-09-04 President and Fellows of Harvard College Evaluation and improvement of nuclease cleavage specificity
US10967298B2 (en) 2012-03-15 2021-04-06 Flodesign Sonics, Inc. Driver and control for variable impedence load
US9745548B2 (en) 2012-03-15 2017-08-29 Flodesign Sonics, Inc. Acoustic perfusion devices
US10322949B2 (en) 2012-03-15 2019-06-18 Flodesign Sonics, Inc. Transducer and reflector configurations for an acoustophoretic device
US10704021B2 (en) 2012-03-15 2020-07-07 Flodesign Sonics, Inc. Acoustic perfusion devices
US9950282B2 (en) 2012-03-15 2018-04-24 Flodesign Sonics, Inc. Electronic configuration and control for acoustic standing wave generation
US9458450B2 (en) 2012-03-15 2016-10-04 Flodesign Sonics, Inc. Acoustophoretic separation technology using multi-dimensional standing waves
US10689609B2 (en) 2012-03-15 2020-06-23 Flodesign Sonics, Inc. Acoustic bioreactor processes
US9752113B2 (en) 2012-03-15 2017-09-05 Flodesign Sonics, Inc. Acoustic perfusion devices
US10737953B2 (en) 2012-04-20 2020-08-11 Flodesign Sonics, Inc. Acoustophoretic method for use in bioreactors
US9163284B2 (en) 2013-08-09 2015-10-20 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
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
US9228207B2 (en) 2013-09-06 2016-01-05 President And Fellows Of Harvard College Switchable gRNAs comprising aptamers
US9745569B2 (en) 2013-09-13 2017-08-29 Flodesign Sonics, Inc. System for generating high concentration factors for low cell density suspensions
US9840699B2 (en) 2013-12-12 2017-12-12 President And Fellows Of Harvard College Methods for nucleic acid editing
EP3092049A1 (en) 2014-01-08 2016-11-16 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
US10077453B2 (en) 2014-07-30 2018-09-18 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
WO2016073990A2 (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
US11708572B2 (en) 2015-04-29 2023-07-25 Flodesign Sonics, Inc. Acoustic cell separation techniques and processes
US11021699B2 (en) 2015-04-29 2021-06-01 FioDesign Sonics, Inc. Separation using angled acoustic waves
US11377651B2 (en) 2016-10-19 2022-07-05 Flodesign Sonics, Inc. Cell therapy processes utilizing acoustophoresis
AU2016291778B2 (en) 2015-07-13 2021-05-06 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
US10166255B2 (en) 2015-07-31 2019-01-01 Regents Of The University Of Minnesota Intracellular genomic transplant and methods of therapy
CA2999500A1 (en) 2015-09-24 2017-03-30 Editas Medicine, Inc. Use of exonucleases to improve crispr/cas-mediated genome editing
CA3001008A1 (en) 2015-10-05 2017-04-13 Precision Biosciences, Inc. Engineered meganucleases with recognition sequences found in the human t cell receptor alpha constant region gene
ES2920525T3 (es) 2015-10-05 2022-08-05 Prec Biosciences Inc Células genéticamente modificadas que comprenden un gen de región constante alfa del receptor de linfocitos T humano modificado
SG10202104041PA (en) 2015-10-23 2021-06-29 Harvard College Nucleobase editors and uses thereof
KR102766526B1 (ko) 2015-10-28 2025-02-13 상가모 테라퓨틱스, 인코포레이티드 간-특이적 작제물인, 인자 viii 발현 카세트 및 이의 사용 방법
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
AU2017250769B2 (en) * 2016-04-15 2022-07-14 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
KR102827276B1 (ko) 2016-08-03 2025-07-01 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 아데노신 핵염기 편집제 및 그의 용도
WO2018031683A1 (en) 2016-08-09 2018-02-15 President And Fellows Of Harvard College Programmable cas9-recombinase fusion proteins and uses thereof
KR20240144493A (ko) * 2016-08-24 2024-10-02 상가모 테라퓨틱스, 인코포레이티드 가공된 표적 특이적 뉴클레아제
WO2018039438A1 (en) 2016-08-24 2018-03-01 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
ES2900317T3 (es) 2016-08-24 2022-03-16 Sangamo Therapeutics Inc Regulación de la expresión génica al usar nucleasas genomanipuladas
MA46354A (fr) 2016-10-03 2019-08-07 Juno Therapeutics Inc Molécules se liant spécifiquement au vph
CN110214180A (zh) 2016-10-14 2019-09-06 哈佛大学的校长及成员们 核碱基编辑器的aav递送
GB2604416B (en) 2016-10-18 2023-03-15 Univ Minnesota Tumor infiltating lymphocytes and methods of therapy
CN110494543A (zh) 2016-10-19 2019-11-22 弗洛设计声能学公司 通过声学的亲和细胞提取
US20200087681A1 (en) * 2016-12-21 2020-03-19 Ucl Business Plc Therapeutic cells
US10745677B2 (en) 2016-12-23 2020-08-18 President And Fellows Of Harvard College Editing of CCR5 receptor gene to protect against HIV infection
JP2020505044A (ja) * 2017-01-26 2020-02-20 サンガモ セラピューティクス, インコーポレイテッド B細胞操作
WO2018165504A1 (en) 2017-03-09 2018-09-13 President And Fellows Of Harvard College Suppression of pain by gene editing
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
KR20190127797A (ko) 2017-03-10 2019-11-13 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 시토신에서 구아닌으로의 염기 편집제
GB2575930A (en) 2017-03-23 2020-01-29 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
KR20240027153A (ko) 2017-06-15 2024-02-29 더 리전트 오브 더 유니버시티 오브 캘리포니아 표적화된 비-바이러스 dna 삽입
JP7419073B2 (ja) * 2017-06-16 2024-01-22 サンガモ セラピューティクス, インコーポレイテッド T細胞および/またはhla受容体の標的化破壊
JP2020530307A (ja) 2017-06-30 2020-10-22 インティマ・バイオサイエンス,インコーポレーテッド 遺伝子治療のためのアデノ随伴ウイルスベクター
WO2019006330A1 (en) * 2017-06-30 2019-01-03 Indiana University Research And Technology Corporation COMPOSITIONS AND METHODS FOR DETECTING REACTIVITY TO ALS
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
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
US11732274B2 (en) 2017-07-28 2023-08-22 President And Fellows Of Harvard College Methods and compositions for evolving base 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
WO2019052577A1 (zh) * 2017-09-18 2019-03-21 博雅辑因(北京)生物科技有限公司 一种基因编辑t细胞及其用途
JP7449223B2 (ja) 2017-09-22 2024-03-13 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア ムコ多糖症ii型を治療するための遺伝子療法
CN111954679A (zh) 2017-10-03 2020-11-17 朱诺治疗学股份有限公司 Hpv特异性结合分子
CA3082251A1 (en) 2017-10-16 2019-04-25 The Broad Institute, Inc. Uses of adenosine base editors
AU2018355587B2 (en) 2017-10-27 2023-02-02 The Regents Of The University Of California Targeted replacement of endogenous T cell receptors
AU2018383712B2 (en) * 2017-12-11 2025-09-04 Editas Medicine, Inc. Cpf1-related methods and compositions for gene editing
JP2021507561A (ja) 2017-12-14 2021-02-22 フロデザイン ソニックス, インク.Flodesign Sonics, Inc. 音響トランスデューサドライバ及びコントローラ
EP3724214A4 (en) 2017-12-15 2021-09-01 The Broad Institute Inc. SYSTEMS AND METHODS FOR PREDICTING REPAIR RESULTS IN GENETIC ENGINEERING
CA3095084A1 (en) 2018-04-05 2019-10-10 Juno Therapeutics, Inc. T cells expressing a recombinant receptor, related polynucleotides and methods
MX2020010459A (es) 2018-04-05 2021-01-20 Juno Therapeutics Inc Metodos para producir celulas que expresan un receptor recombinante y composiciones relacionadas.
MX2020010460A (es) 2018-04-05 2021-01-29 Juno Therapeutics Inc Receptores de células t, y células diseñadas que expresan los mismos.
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
WO2019226953A1 (en) 2018-05-23 2019-11-28 The Broad Institute, Inc. Base editors and uses thereof
EP3814492A4 (en) 2018-06-29 2022-02-23 Wuhan Neurophth Biotechnology Limited Company COMPOSITIONS AND METHODS OF TREATING LEBER'S HEREDITARY OPTIC NEUROPATHY
AU2019301053A1 (en) 2018-07-09 2021-01-28 Flagship Pioneering Innovations V, Inc. Fusosome compositions and uses thereof
CA3105382A1 (en) 2018-08-03 2020-02-06 Sangamo Therapeutics, Inc. Improved clinical parameters by expression of factor viii
EP3833752A4 (en) * 2018-08-07 2022-06-01 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
GB201813528D0 (en) 2018-08-20 2018-10-03 Ucl Business Plc Factor IX encoding nucleotides
SG11202101032VA (en) 2018-08-20 2021-02-25 Wuhan Neurophth Biotechnology Ltd 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
WO2020038352A1 (en) * 2018-08-20 2020-02-27 Wuhan Neurophth Biological Technology Limited Company Compositions and methods for treating leber's hereditary optic neuropathy
WO2020082046A2 (en) 2018-10-18 2020-04-23 Intellia Therapeutics, Inc. Compositions and methods for expressing factor ix
US12281338B2 (en) 2018-10-29 2025-04-22 The Broad Institute, Inc. Nucleobase editors comprising GeoCas9 and uses thereof
EP3880831A1 (en) 2018-11-14 2021-09-22 Flagship Pioneering Innovations V, Inc. Compositions and methods for compartment-specific cargo delivery
JP2022507453A (ja) * 2018-11-14 2022-01-18 フラッグシップ パイオニアリング イノベーションズ ブイ, インコーポレイテッド T細胞送達のためのフソソーム組成物
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
CA3106693A1 (en) 2019-02-06 2020-08-13 Sangamo Therapeutics, Inc. Method for the treatment of mucopolysaccharidosis type i
DE112020001306T5 (de) 2019-03-19 2022-01-27 Massachusetts Institute Of Technology Verfahren und zusammensetzungen zur editierung von nukleotidsequenzen
US12473543B2 (en) 2019-04-17 2025-11-18 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
SG11202111372VA (en) 2019-05-01 2021-11-29 Juno Therapeutics Inc Cells expressing a chimeric receptor from a modified cd247 locus, related polynucleotides and methods
US20220184131A1 (en) 2019-05-01 2022-06-16 Juno Therapeutics, Inc. Cells expressing a recombinant receptor from a modified tgfbr2 locus, related polynucleotides and methods
SG11202111256XA (en) 2019-06-07 2021-11-29 Regeneron Pharma Non-human animals comprising a humanized albumin locus
EP4004052A4 (en) * 2019-07-30 2023-11-01 Provention Bio, Inc. METHODS AND COMPOSITIONS FOR REDUCING IMMUNOGENICITY BY NON-DEPLETED B CELL INHIBITORS
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
AU2021267940A1 (en) 2020-05-08 2022-12-08 President And Fellows Of Harvard College Methods and compositions for simultaneous editing of both strands of a target double-stranded nucleotide sequence
KR20230022868A (ko) 2020-05-13 2023-02-16 주노 쎄러퓨티크스 인코퍼레이티드 재조합 수용체를 발현하는 공여자-배치 세포의 제조 방법
CN116234558A (zh) 2020-06-26 2023-06-06 朱诺治疗学有限公司 条件性地表达重组受体的工程化t细胞、相关多核苷酸和方法
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
PE20241173A1 (es) 2021-10-14 2024-05-28 Arsenal Biosciences Inc Celulas inmunitarias que tienen arnhc coexpresados y sistemas de compuerta logica
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
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
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

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
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
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
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
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
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
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
ES2316148T3 (es) 1994-03-23 2009-04-01 Ohio University Acidos nucleicos compactados y su suministro a celulas.
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
USRE45721E1 (en) 1994-08-20 2015-10-06 Gendaq, Ltd. Relating to binding proteins for recognition of DNA
US5789538A (en) 1995-02-03 1998-08-04 Massachusetts Institute Of Technology Zinc finger proteins with high affinity new DNA binding specificities
US5925523A (en) 1996-08-23 1999-07-20 President & Fellows Of Harvard College Intraction trap assay, reagents and uses thereof
GB9703369D0 (en) 1997-02-18 1997-04-09 Lindqvist Bjorn H Process
GB2338237B (en) 1997-02-18 2001-02-28 Actinova Ltd In vitro peptide or protein expression library
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
ATE466952T1 (de) 1998-03-02 2010-05-15 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
ATE451389T1 (de) * 1998-11-10 2009-12-15 Ludwig Inst Cancer Res Von blutplättchen abstammender wachstumsfaktor d, dafür kodierende dns und deren verwendungen
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
US6503717B2 (en) 1999-12-06 2003-01-07 Sangamo Biosciences, Inc. Methods of using randomized libraries of zinc finger proteins for the identification of gene function
US6599692B1 (en) 1999-09-14 2003-07-29 Sangamo Bioscience, Inc. Functional genomics using zinc finger proteins
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
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
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
US20030044787A1 (en) 2000-05-16 2003-03-06 Joung J. Keith Methods and compositions for interaction trap assays
CA2398687C (en) 2000-05-16 2008-09-23 Comsonics, Inc. Device and method of determining location of signal ingress
JP2002060786A (ja) 2000-08-23 2002-02-26 Kao Corp 硬質表面用殺菌防汚剤
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
WO2003016496A2 (en) 2001-08-20 2003-02-27 The Scripps Research Institute 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
CN100575485C (zh) 2002-01-23 2009-12-30 犹他大学研究基金会 使用锌指核酸酶的定向染色体诱变
EP2368982A3 (en) 2002-03-21 2011-10-12 Sangamo BioSciences, Inc. 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
JP2006502748A (ja) 2002-09-05 2006-01-26 カリフォルニア インスティテュート オブ テクノロジー 遺伝子ターゲッティングを誘発するキメラヌクレアーゼの使用方法
GB0312086D0 (en) * 2003-05-27 2003-07-02 Novartis Ag Organic compounds
US8409861B2 (en) 2003-08-08 2013-04-02 Sangamo Biosciences, Inc. Targeted deletion of cellular DNA sequences
US7888121B2 (en) 2003-08-08 2011-02-15 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
US7972854B2 (en) 2004-02-05 2011-07-05 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
WO2005099771A2 (en) 2004-04-08 2005-10-27 Sangamo Biosciences, Inc. Methods and compositions for treating neuropathic and neurodegenerative conditions
AU2005233550B2 (en) 2004-04-08 2010-11-18 Sangamo Therapeutics, Inc. Treatment of neuropathic pain with zinc finger proteins
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
EP1789095A2 (en) 2004-09-16 2007-05-30 Sangamo Biosciences Inc. Compositions and methods for protein production
MX2007013757A (es) 2005-05-05 2008-01-24 Univ Arizona Reagrupacion permitida de secuencia (seer)-met odo de novedad para visualizar secuencias de adn especificas.
WO2007014275A2 (en) 2005-07-26 2007-02-01 Sangamo Biosciences, Inc. Targeted integration and expression of exogenous nucleic acid sequences
ATE549401T1 (de) 2005-10-18 2012-03-15 Prec Biosciences Rational konstruierte meganukleasen mit veränderter sequenzspezifität und dna- bindungsaffinität
EP2019839B1 (en) 2006-05-25 2011-12-07 Sangamo BioSciences, Inc. Methods and compositions for gene inactivation
CA2651494C (en) 2006-05-25 2015-09-29 Sangamo Biosciences, Inc. Engineered cleavage half-domains
EP2137310B1 (en) 2007-04-26 2010-11-24 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
JP2011518555A (ja) 2008-04-14 2011-06-30 サンガモ バイオサイエンシーズ, インコーポレイテッド 標的組込みのための線形ドナーコンストラクト
KR20160015400A (ko) 2008-08-22 2016-02-12 상가모 바이오사이언스 인코포레이티드 표적화된 단일가닥 분할 및 표적화된 통합을 위한 방법 및 조성물
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
CA2745031C (en) 2008-12-04 2018-08-14 Sangamo Biosciences, Inc. Genome editing in rats using zinc-finger nucleases
BRPI0922641B1 (pt) 2008-12-17 2021-10-26 Dow Agrosciences Llc Método de integração de uma ou mais sequências de ácido nucleico exógenas no genoma de uma célula vegetal
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
AU2011213242B2 (en) 2010-02-08 2015-01-29 Sangamo Therapeutics, Inc. Engineered cleavage half-domains
JP2013518602A (ja) 2010-02-09 2013-05-23 サンガモ バイオサイエンシーズ, インコーポレイテッド 部分的に一本鎖のドナー分子による標的化ゲノム改変
WO2011123842A2 (en) * 2010-04-02 2011-10-06 Ceregene, Inc. Methods for treating parkinson's disease and other disorders of dopaminergic neurons of the brain
KR101880536B1 (ko) 2010-04-26 2018-07-23 상가모 테라퓨틱스, 인코포레이티드 아연 핑거 뉴클레아제를 사용하는 로사 좌위의 게놈 편집
WO2011139349A1 (en) 2010-05-03 2011-11-10 Sangamo Biosciences, Inc. Compositions for linking zinc finger modules
WO2011146121A1 (en) 2010-05-17 2011-11-24 Sangamo Biosciences, Inc. Novel dna-binding proteins and uses thereof
CA2805442C (en) 2010-07-21 2020-05-12 Sangamo Biosciences, Inc. Methods and compositions for modification of an hla locus
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
EP2758529B1 (en) 2011-09-21 2019-03-20 Sangamo Therapeutics, Inc. Methods and compositions for regulation of transgene expression
AU2012328682B2 (en) 2011-10-27 2017-09-21 Sangamo Therapeutics, Inc. Methods and compositions for modification of the HPRT locus
HK1200871A1 (en) 2011-11-16 2015-08-14 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
JP6463672B2 (ja) * 2012-05-25 2019-02-06 セレクティスCellectis Tcrアルファ欠損t細胞を増殖させるためのプレtアルファまたはその機能性変種の使用
US10648001B2 (en) 2012-07-11 2020-05-12 Sangamo Therapeutics, Inc. Method of treating mucopolysaccharidosis type I or II
JP6401704B2 (ja) 2012-10-10 2018-10-10 サンガモ セラピューティクス, インコーポレイテッド T細胞を修飾する化合物およびその使用
HK1215873A1 (zh) 2012-12-05 2016-09-23 桑格摩治疗股份有限公司 調整代謝紊亂的方法及組合物
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
CA2910489A1 (en) 2013-05-15 2014-11-20 Sangamo Biosciences, Inc. Methods and compositions for treatment of a genetic condition
US9999206B2 (en) 2013-07-25 2018-06-19 Utah State University Model and method for a transgenic bovidae expressing cardiac fibrosis and associated pathology
HUE051628T2 (hu) 2013-12-09 2021-03-01 Sangamo Therapeutics Inc Eljárások és kompozíciók genom-manipulálására
CA2939942A1 (en) 2014-02-24 2015-08-27 Sangamo Biosciences, 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
JP2017515819A (ja) 2014-05-08 2017-06-15 サンガモ セラピューティクス, インコーポレイテッド ハンチントン病を処置するための方法および組成物
US9816074B2 (en) 2014-07-25 2017-11-14 Sangamo Therapeutics, Inc. Methods and compositions for modulating nuclease-mediated genome engineering in hematopoietic stem cells
US9616090B2 (en) 2014-07-30 2017-04-11 Sangamo Biosciences, Inc. Gene correction of SCID-related genes in hematopoietic stem and progenitor cells
AU2016291778B2 (en) 2015-07-13 2021-05-06 Sangamo Therapeutics, Inc. Delivery methods and compositions for nuclease-mediated genome engineering
ES2920525T3 (es) * 2015-10-05 2022-08-05 Prec Biosciences Inc Células genéticamente modificadas que comprenden un gen de región constante alfa del receptor de linfocitos T humano modificado
KR102766526B1 (ko) 2015-10-28 2025-02-13 상가모 테라퓨틱스, 인코포레이티드 간-특이적 작제물인, 인자 viii 발현 카세트 및 이의 사용 방법

Also Published As

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

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