CN112955557A - 用于治疗丙酸血症的基因疗法 - Google Patents

用于治疗丙酸血症的基因疗法 Download PDF

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Publication number
CN112955557A
CN112955557A CN201980070896.1A CN201980070896A CN112955557A CN 112955557 A CN112955557 A CN 112955557A CN 201980070896 A CN201980070896 A CN 201980070896A CN 112955557 A CN112955557 A CN 112955557A
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sequence
raav
pcca
pccb
seq
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马修·斯科特·福勒
塞缪尔·沃兹沃斯
凯利里德·克拉克
肖恩·克里斯托弗·多尔蒂
斯图尔特·克雷格
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Ultragenyx Pharmaceutical Inc
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    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y604/00—Ligases forming carbon-carbon bonds (6.4)
    • C12Y604/01—Ligases forming carbon-carbon bonds (6.4.1)
    • C12Y604/01003—Propionyl-CoA carboxylase (6.4.1.3)
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/072—Animals genetically altered by homologous recombination maintaining or altering function, i.e. knock in
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
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    • C12N2830/00—Vector systems having a special element relevant for transcription
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    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6876—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes
    • C12Q1/6883—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material

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  • Proteomics, Peptides & Aminoacids (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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CN201980070896.1A 2018-10-01 2019-10-01 用于治疗丙酸血症的基因疗法 Pending CN112955557A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862739471P 2018-10-01 2018-10-01
US62/739,471 2018-10-01
PCT/US2019/054003 WO2020072451A1 (en) 2018-10-01 2019-10-01 Gene therapy for treating propionic acidemia

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CN112955557A true CN112955557A (zh) 2021-06-11

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US (2) US12472268B2 (https=)
EP (1) EP3861131A4 (https=)
JP (1) JP2022513318A (https=)
KR (1) KR20210082460A (https=)
CN (1) CN112955557A (https=)
AR (1) AR116569A1 (https=)
AU (1) AU2019354296A1 (https=)
BR (1) BR112021006052A2 (https=)
CA (1) CA3113975A1 (https=)
TW (1) TW202028472A (https=)
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WO2021076797A1 (en) * 2019-10-15 2021-04-22 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Synthetic genes for the treatment of propionic acidemia caused by mutations in propionyl-coa carboxylase beta
WO2021202532A1 (en) 2020-03-31 2021-10-07 Ultragenyx Pharmaceutical Inc. Gene therapy for treating propionic acidemia
EP4308174A4 (en) * 2021-03-19 2025-07-30 Mayo Found Medical Education & Res METHODS AND MATERIALS FOR THE TREATMENT OF PROPIONIC ACIDEMIA
WO2023130003A2 (en) * 2021-12-29 2023-07-06 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Improved gene therapy constructs for the treatment of propionic acidemia caused by mutations in propionyl-coa carboxylase alpha

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Publication number Priority date Publication date Assignee Title
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US12472268B2 (en) 2025-11-18
JP2022513318A (ja) 2022-02-07
KR20210082460A (ko) 2021-07-05
US20210283272A1 (en) 2021-09-16
AU2019354296A1 (en) 2021-04-29
WO2020072451A1 (en) 2020-04-09
TW202028472A (zh) 2020-08-01
EP3861131A1 (en) 2021-08-11
CA3113975A1 (en) 2020-04-09
EP3861131A4 (en) 2022-06-15
BR112021006052A2 (pt) 2021-09-08
WO2020072451A8 (en) 2021-05-14
AR116569A1 (es) 2021-05-19
US20260041788A1 (en) 2026-02-12

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