CN112888777A - 用于造血细胞的基因修饰的方法 - Google Patents

用于造血细胞的基因修饰的方法 Download PDF

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Publication number
CN112888777A
CN112888777A CN201980049019.6A CN201980049019A CN112888777A CN 112888777 A CN112888777 A CN 112888777A CN 201980049019 A CN201980049019 A CN 201980049019A CN 112888777 A CN112888777 A CN 112888777A
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cells
poloxamer
pge2
leu
cell
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CN201980049019.6A
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Chinese (zh)
Inventor
B·比尔德
G·D·沙
J·A·布伦罗恩塞洛
J·C·塞格瓦桑兹
P·瑞欧加尔道
S·纳瓦罗奥尔多纳兹
E·阿尔玛尔扎诺瓦
O·奎恩塔纳布斯塔曼特
C·迈萨努纳兹
K·劳
K·帕特尔
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Biomedical Network Research Consortium Center MP
Centro de Investigaciones Energeticas Medioambientales y Tecnologicas CIEMAT
Instituto de Investigacion Sanitaria Fundacion Jimenez Diaz
Rocket Pharmaceuticals Inc
Original Assignee
Biomedical Network Research Consortium Center MP
Centro de Investigaciones Energeticas Medioambientales y Tecnologicas CIEMAT
Instituto de Investigacion Sanitaria Fundacion Jimenez Diaz
Rocket Pharmaceuticals Inc
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Application filed by Biomedical Network Research Consortium Center MP, Centro de Investigaciones Energeticas Medioambientales y Tecnologicas CIEMAT, Instituto de Investigacion Sanitaria Fundacion Jimenez Diaz, Rocket Pharmaceuticals Inc filed Critical Biomedical Network Research Consortium Center MP
Publication of CN112888777A publication Critical patent/CN112888777A/zh
Pending legal-status Critical Current

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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/28—Bone marrow; Haematopoietic stem cells; Mesenchymal stem cells of any origin, e.g. adipose-derived stem cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/06—Antianaemics
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0634—Cells from the blood or the immune system
    • C12N5/0647—Haematopoietic stem cells; Uncommitted or multipotent progenitors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2500/00—Specific components of cell culture medium
    • C12N2500/50—Soluble polymers, e.g. polyethyleneglycol [PEG]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2510/00—Genetically modified cells
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
    • C12N2740/15041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/15043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Hematology (AREA)
  • Organic Chemistry (AREA)
  • Developmental Biology & Embryology (AREA)
  • Immunology (AREA)
  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Cell Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Veterinary Medicine (AREA)
  • Microbiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biochemistry (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Virology (AREA)
  • Medicinal Chemistry (AREA)
  • Epidemiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Diabetes (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Molecular Biology (AREA)
  • Plant Pathology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
CN201980049019.6A 2018-07-30 2019-07-30 用于造血细胞的基因修饰的方法 Pending CN112888777A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862712146P 2018-07-30 2018-07-30
US62/712,146 2018-07-30
PCT/US2019/044237 WO2020028430A1 (en) 2018-07-30 2019-07-30 Methods for gene modification of hematopoietic cells

Publications (1)

Publication Number Publication Date
CN112888777A true CN112888777A (zh) 2021-06-01

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CN201980049019.6A Pending CN112888777A (zh) 2018-07-30 2019-07-30 用于造血细胞的基因修饰的方法

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US (1) US20210290685A1 (https=)
EP (1) EP3830248A4 (https=)
JP (2) JP7664829B2 (https=)
KR (1) KR20210046004A (https=)
CN (1) CN112888777A (https=)
AU (1) AU2019315438B2 (https=)
BR (1) BR112021001757A2 (https=)
CA (1) CA3106241A1 (https=)
IL (2) IL280465B2 (https=)
MX (1) MX2021001292A (https=)
SG (1) SG11202100025QA (https=)
WO (1) WO2020028430A1 (https=)

Families Citing this family (9)

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HUE055862T2 (hu) 2016-04-20 2021-12-28 Centro De Investig Energeticas Készítmények és eljárások PKLR fokozott génexpressziójára
SG11202002263TA (en) 2017-10-16 2020-04-29 Centro De Investigaciones Energeticas Medioambientales Y Tecnologicas Lentiviral vectors for delivery of pklr to treat pyruvate kinase deficiency
WO2019200167A1 (en) 2018-04-11 2019-10-17 Rocket Pharmaceuticals, Ltd. Compositions and methods for stem cell transplant
MX2020011386A (es) 2018-04-27 2021-01-29 Spacecraft Seven Llc Terapia genica para la degeneracion del snc.
CN115243705A (zh) * 2020-05-27 2022-10-25 苏黎世大学 新颖的转导增强剂及其用途
KR20230043869A (ko) 2020-08-07 2023-03-31 스페이스크래프트 세븐, 엘엘씨 Aav 벡터를 사용한 플라코필린-2(pkp2) 유전자 요법
CN114763563B (zh) * 2021-01-12 2024-04-30 深圳华大临床检验中心 一种提高慢病毒感染效率的方法
AU2022264498A1 (en) 2021-04-26 2023-09-28 Strm.Bio Incorporated Compositions and methods related to megakaryocyte-derived extracellular vesicles for fanconi anemia
WO2025106604A1 (en) * 2023-11-14 2025-05-22 The University Of North Carolina At Chapel Hill Compositions and methods relating to antiviral therapeutics

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2017139576A1 (en) * 2016-02-12 2017-08-17 Bluebird Bio, Inc. Vcn enhancer compositions and methods of using the same
WO2017218519A1 (en) * 2016-06-13 2017-12-21 Bluebird Bio, Inc. Gene therapy of neuronal ceroid lipofuscinoses
WO2018106821A1 (en) * 2016-12-06 2018-06-14 Bluebird Bio, Inc. Gene therapy for mucopolysaccharidosis, type ii
WO2018106807A1 (en) * 2016-12-06 2018-06-14 Bluebird Bio, Inc. Gene therapy for mucopolysaccharidosis, type i

Family Cites Families (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20240151726A (ko) * 2016-09-08 2024-10-18 센트로 데 인베스띠가씨오네스 에너제티까스 메디오암비엔딸레스 와이 테크놀로지까스, 오.에이., 엠.피. 판코니 빈혈 환자를 위한 유전자 치료

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2017139576A1 (en) * 2016-02-12 2017-08-17 Bluebird Bio, Inc. Vcn enhancer compositions and methods of using the same
WO2017218519A1 (en) * 2016-06-13 2017-12-21 Bluebird Bio, Inc. Gene therapy of neuronal ceroid lipofuscinoses
WO2018106821A1 (en) * 2016-12-06 2018-06-14 Bluebird Bio, Inc. Gene therapy for mucopolysaccharidosis, type ii
WO2018106807A1 (en) * 2016-12-06 2018-06-14 Bluebird Bio, Inc. Gene therapy for mucopolysaccharidosis, type i

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
JOHN F. DIPERSIO: "Phase III Prospective Randomized Double-Blind Placebo-Controlled Trial of Plerixafor Plus Granulocyte Colony-Stimulating Factor Compared With Placebo Plus Granulocyte Colony-Stimulating Factor for Autologous Stem-Cell Mobilization and Transplantation for Patients With Non-Hodgkin’s Lymphoma", JOURNAL OF CLINICAL ONCOLOGY, vol. 27, no. 28, 31 December 2009 (2009-12-31), pages 4767 - 4773, XP093040108, DOI: 10.1200/JCO.2008.20.7209 *
STEPHEN J.FORMAN: "THOMAS\'HEMATOPOIETIC CELL TRANSPLANTATION：stem cell transplantation", vol. 5, 31 December 2016, pages: 82 - 83 *

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Publication number Publication date
SG11202100025QA (en) 2021-01-28
IL280465B2 (en) 2025-03-01
MX2021001292A (es) 2021-07-15
EP3830248A1 (en) 2021-06-09
US20210290685A1 (en) 2021-09-23
JP2021532776A (ja) 2021-12-02
WO2020028430A1 (en) 2020-02-06
JP2025038075A (ja) 2025-03-18
IL280465B1 (en) 2024-11-01
AU2019315438B2 (en) 2026-02-05
AU2019315438A1 (en) 2021-01-28
JP7664829B2 (ja) 2025-04-18
IL316185A (en) 2024-12-01
IL280465A (en) 2021-03-25
EP3830248A4 (en) 2022-05-04
KR20210046004A (ko) 2021-04-27
BR112021001757A2 (pt) 2021-05-11
CA3106241A1 (en) 2020-02-06

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