KR20210046004A - 조혈 세포의 유전자 변형 방법 - Google Patents

조혈 세포의 유전자 변형 방법 Download PDF

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Publication number
KR20210046004A
KR20210046004A KR1020217005904A KR20217005904A KR20210046004A KR 20210046004 A KR20210046004 A KR 20210046004A KR 1020217005904 A KR1020217005904 A KR 1020217005904A KR 20217005904 A KR20217005904 A KR 20217005904A KR 20210046004 A KR20210046004 A KR 20210046004A
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South Korea
Prior art keywords
cells
poloxamer
pge2
leu
hematopoietic cells
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KR1020217005904A
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English (en)
Korean (ko)
Inventor
브라이언 비어드
가우라브 디. 샤
론세로 후안 안토니오 부에렌
산즈 호세 카를로스 세고비아
갈도 파울라 리오
올도네즈 수산나 나바로
노보아 엘레나 알마르자
부스타만트 오스카 퀸타나
누네즈 크리스티나 메사
케네스 로
키나리 파텔
Original Assignee
센트로 데 인베스띠가씨오네스 에너제티까스 메디오암비엔딸레스 와이 테크놀로지까스, 오.에이., 엠.피.
푼다시안 인스티튜토 데 인베스티카시온 사니타리아 푼다시안 시메네스 디아즈
로켓 파마슈티컬스, 리미티드
콘소르시오 센트로 데 인베스티카시온 비오메디카 엔 레드, 엠.피.
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Application filed by 센트로 데 인베스띠가씨오네스 에너제티까스 메디오암비엔딸레스 와이 테크놀로지까스, 오.에이., 엠.피., 푼다시안 인스티튜토 데 인베스티카시온 사니타리아 푼다시안 시메네스 디아즈, 로켓 파마슈티컬스, 리미티드, 콘소르시오 센트로 데 인베스티카시온 비오메디카 엔 레드, 엠.피. filed Critical 센트로 데 인베스띠가씨오네스 에너제티까스 메디오암비엔딸레스 와이 테크놀로지까스, 오.에이., 엠.피.
Publication of KR20210046004A publication Critical patent/KR20210046004A/ko
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)
KR1020217005904A 2018-07-30 2019-07-30 조혈 세포의 유전자 변형 방법 Pending KR20210046004A (ko)

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
KR20210046004A true KR20210046004A (ko) 2021-04-27

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KR1020217005904A Pending KR20210046004A (ko) 2018-07-30 2019-07-30 조혈 세포의 유전자 변형 방법

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Country Link
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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Publication number Priority date Publication date Assignee Title
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

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* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
IL261002B2 (en) * 2016-02-12 2024-07-01 Bluebird Bio Inc Vcn enhancer compositions and methods of using the same
CA3058735A1 (en) 2016-06-13 2017-12-21 Bluebird Bio, Inc. Gene therapy of neuronal ceroid lipofuscinoses
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
BR112019011635A2 (pt) 2016-12-06 2019-11-12 Bluebird Bio Inc terapia genética para mucopolissacaridose, tipo i
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

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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
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
JP7664829B2 (ja) 2025-04-18
IL316185A (en) 2024-12-01
IL280465A (en) 2021-03-25
EP3830248A4 (en) 2022-05-04
BR112021001757A2 (pt) 2021-05-11
CA3106241A1 (en) 2020-02-06

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