JP2023500793A - アルツハイマー病に対する遺伝子治療方法 - Google Patents

アルツハイマー病に対する遺伝子治療方法 Download PDF

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JP2023500793A
JP2023500793A JP2022522814A JP2022522814A JP2023500793A JP 2023500793 A JP2023500793 A JP 2023500793A JP 2022522814 A JP2022522814 A JP 2022522814A JP 2022522814 A JP2022522814 A JP 2022522814A JP 2023500793 A JP2023500793 A JP 2023500793A
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ロナルド ジー． クリスタル
ケイティー エム． スタイルズ
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Cornell University
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    • 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
    • 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
    • A01K2217/05—Animals comprising random inserted nucleic acids (transgenic)
    • A01K2217/052—Animals comprising random inserted nucleic acids (transgenic) inducing gain of function
    • 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
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • 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
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
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    • A01K2267/0312—Animal model for Alzheimer's disease
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C12N2310/141—MicroRNAs, miRNAs
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    • C12N2310/50—Physical structure
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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
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
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JP2022522814A 2019-10-16 2020-10-16 アルツハイマー病に対する遺伝子治療方法 Pending JP2023500793A (ja)

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JP2025233417A JP2026041903A (ja) 2019-10-16 2025-12-05 アルツハイマー病に対する遺伝子治療方法

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US201962915988P 2019-10-16 2019-10-16
US62/915,988 2019-10-16
PCT/US2020/056051 WO2021076941A1 (en) 2019-10-16 2020-10-16 Gene therapy for alzheimer's disease

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JP2023500793A5 JP2023500793A5 (https=) 2023-10-24
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US (1) US20230405148A1 (https=)
EP (1) EP4045653A1 (https=)
JP (2) JP2023500793A (https=)
KR (1) KR20220082050A (https=)
CN (1) CN114761569A (https=)
AU (2) AU2020367437B2 (https=)
BR (1) BR112022007010A2 (https=)
CA (1) CA3157864A1 (https=)
CO (1) CO2022006367A2 (https=)
IL (1) IL292148A (https=)
MX (1) MX2022004524A (https=)
WO (1) WO2021076941A1 (https=)
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Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US12611467B2 (en) 2020-11-25 2026-04-28 Cornell University APOE gene therapy

Families Citing this family (4)

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Publication number Priority date Publication date Assignee Title
JP7616995B2 (ja) * 2018-11-28 2025-01-17 プリベイル セラピューティクス，インコーポレーテッド 神経変性疾患のための遺伝子治療
EP4508213A1 (en) * 2022-04-12 2025-02-19 uniQure biopharma B.V. Nucleic acid regulation of apoe
EP4551711A1 (en) * 2022-07-08 2025-05-14 Cornell University Methods and pharmaceutical compositions for the treatment and the prevention of alzheimers disease
WO2025213111A2 (en) * 2024-04-05 2025-10-09 Cornell University Aav expression of mirna to suppress human apoe mrna

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Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US12611467B2 (en) 2020-11-25 2026-04-28 Cornell University APOE gene therapy

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US20230405148A1 (en) 2023-12-21
CA3157864A1 (en) 2021-04-22
JP2026041903A (ja) 2026-03-10
WO2021076941A1 (en) 2021-04-22
ZA202205344B (en) 2023-03-29
AU2020367437B2 (en) 2024-11-07
BR112022007010A2 (pt) 2022-07-12
AU2025200812A1 (en) 2025-02-27
MX2022004524A (es) 2022-07-21
EP4045653A1 (en) 2022-08-24
WO2021076941A9 (en) 2021-10-07
KR20220082050A (ko) 2022-06-16
IL292148A (en) 2022-06-01
AU2020367437A1 (en) 2022-06-02
CO2022006367A2 (es) 2022-08-09
CN114761569A (zh) 2022-07-15

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