JP7664829B2 - 造血細胞の遺伝子改変のための方法 - Google Patents

造血細胞の遺伝子改変のための方法 Download PDF

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JP7664829B2
JP7664829B2 JP2021505315A JP2021505315A JP7664829B2 JP 7664829 B2 JP7664829 B2 JP 7664829B2 JP 2021505315 A JP2021505315 A JP 2021505315A JP 2021505315 A JP2021505315 A JP 2021505315A JP 7664829 B2 JP7664829 B2 JP 7664829B2
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poloxamer
pge2
hematopoietic
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JP2021532776A (ja
JP2021532776A5 (enExample
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ブライアン ビアード
ゴーラヴ ディー． シャー
ロンセロ フアン アントニオ ブエレン
サンス ホセ カルロス セゴビア
ガルド パウラ リオ
オルドネス スサナ ナバーロ
ノボア エレナ アルマルサ
ブスタマンテ オスカー キンタナ
ヌニェス クリスティーナ メサ
ケネス ロウ
キンナリー パテル
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Instituto de Investigacion Sanitaria Fundacion Jimenez Diaz
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Instituto de Investigacion Sanitaria Fundacion Jimenez Diaz
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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
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    • 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)
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  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
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  • Biochemistry (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Virology (AREA)
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  • General Chemical & Material Sciences (AREA)
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  • 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)
JP2021505315A 2018-07-30 2019-07-30 造血細胞の遺伝子改変のための方法 Active JP7664829B2 (ja)

Priority Applications (1)

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JP2024218336A JP2025038075A (ja) 2018-07-30 2024-12-13 造血細胞の遺伝子改変のための方法

Applications Claiming Priority (3)

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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

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JP2021532776A JP2021532776A (ja) 2021-12-02
JP2021532776A5 JP2021532776A5 (enExample) 2022-08-04
JP7664829B2 true JP7664829B2 (ja) 2025-04-18

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KR (1) KR20210046004A (enExample)
CN (1) CN112888777A (enExample)
AU (1) AU2019315438B2 (enExample)
BR (1) BR112021001757A2 (enExample)
CA (1) CA3106241A1 (enExample)
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MX (1) MX2021001292A (enExample)
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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 (3)

* 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
WO2018106807A1 (en) 2016-12-06 2018-06-14 Bluebird Bio, Inc. Gene therapy for mucopolysaccharidosis, type i

Family Cites Families (2)

* 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
JP2020500562A (ja) * 2016-12-06 2020-01-16 ブルーバード バイオ， インコーポレイテッド ムコ多糖症ｉｉ型のための遺伝子治療

Patent Citations (3)

* 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
WO2018106807A1 (en) 2016-12-06 2018-06-14 Bluebird Bio, Inc. Gene therapy for mucopolysaccharidosis, type i

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
RELANDER, T et al.，Retroviral transduction of human CD34+ cells on fbronectin fragment CH-296 is inhibited by high concentrations of vector containing medium，J Gene Med，vol.3, no.3，2001年，pp.207-218

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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
CN112888777A (zh) 2021-06-01
JP2025038075A (ja) 2025-03-18
IL280465B1 (en) 2024-11-01
AU2019315438B2 (en) 2026-02-05
AU2019315438A1 (en) 2021-01-28
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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