CN112153973A - 用于干细胞移植的组合物和方法 - Google Patents

用于干细胞移植的组合物和方法 Download PDF

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Publication number
CN112153973A
CN112153973A CN201980018070.0A CN201980018070A CN112153973A CN 112153973 A CN112153973 A CN 112153973A CN 201980018070 A CN201980018070 A CN 201980018070A CN 112153973 A CN112153973 A CN 112153973A
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cells
cell population
enriched cell
stringency
gene
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Chinese (zh)
Inventor
B·彼尔德
J·塞维拉纳瓦罗
G·萨
K·帕特尔
R·普拉布哈卡
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Fundacion Para La Investigacion Biomedica Del Hospital Infantil Universitario Nino Jesus
Rocket Pharmaceuticals Inc
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Fundacion Para La Investigacion Biomedica Del Hospital Infantil Universitario Nino Jesus
Rocket Pharmaceuticals Inc
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • 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
    • 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
    • A61K35/14—Blood; Artificial blood
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
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    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2510/00—Genetically modified cells
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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/10—Plasmid DNA
    • C12N2800/106—Plasmid DNA for vertebrates
    • C12N2800/107—Plasmid DNA for vertebrates for mammalian
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites

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  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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CN201980018070.0A 2018-04-11 2019-04-11 用于干细胞移植的组合物和方法 Pending CN112153973A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862656292P 2018-04-11 2018-04-11
US62/656,292 2018-04-11
PCT/US2019/027083 WO2019200167A1 (en) 2018-04-11 2019-04-11 Compositions and methods for stem cell transplant

Publications (1)

Publication Number Publication Date
CN112153973A true CN112153973A (zh) 2020-12-29

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US (1) US12539316B2 (https=)
JP (2) JP7576754B2 (https=)
KR (1) KR20210003738A (https=)
CN (1) CN112153973A (https=)
AU (2) AU2019252662A1 (https=)
BR (1) BR112020020841A2 (https=)
CA (1) CA3093708A1 (https=)
IL (2) IL307996A (https=)
MX (1) MX2020010718A (https=)
SG (1) SG11202008400SA (https=)
WO (1) WO2019200167A1 (https=)

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