JP7624410B2 - レット症候群の治療におけるコレステロール 24-ヒドロラーゼのための発現ベクター - Google Patents

レット症候群の治療におけるコレステロール 24-ヒドロラーゼのための発現ベクター Download PDF

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JP7624410B2
JP7624410B2 JP2021568858A JP2021568858A JP7624410B2 JP 7624410 B2 JP7624410 B2 JP 7624410B2 JP 2021568858 A JP2021568858 A JP 2021568858A JP 2021568858 A JP2021568858 A JP 2021568858A JP 7624410 B2 JP7624410 B2 JP 7624410B2
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composition
vector
mecp2
aav
cyp46a1
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JP2022534202A5 (https=
JP2022534202A (ja
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ピゲ,フランソワーズ
カルティエ-ラカーヴ,ナタリー
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Centre National de la Recherche Scientifique CNRS
Sorbonne Universite
Institut du Cerveau et de La Moelle Epiniere ICM
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Sorbonne Universite
Institut du Cerveau et de La Moelle Epiniere ICM
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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
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    • A61K38/46Hydrolases (3)
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • C12N9/0004Oxidoreductases (1.)
    • C12N9/0071Oxidoreductases (1.) acting on paired donors with incorporation of molecular oxygen (1.14)
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    • C12N9/50Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
    • C12N9/64Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
    • C12N9/6421Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue from mammals
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    • C12Y114/13Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14) with NADH or NADPH as one donor, and incorporation of one atom of oxygen (1.14.13)
    • C12Y114/13098Cholesterol 24-hydroxylase (1.14.13.98)
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    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
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    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
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    • C12N2800/106Plasmid DNA for vertebrates
    • C12N2800/107Plasmid DNA for vertebrates for mammalian

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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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JP2021568858A 2019-05-21 2020-05-20 レット症候群の治療におけるコレステロール 24-ヒドロラーゼのための発現ベクター Active JP7624410B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP19175767.3 2019-05-21
EP19175767 2019-05-21
PCT/EP2020/064092 WO2020234363A2 (en) 2019-05-21 2020-05-20 Expression vector for cholesterol 24-hydrolase in therapy of rett syndrome

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JP2022534202A JP2022534202A (ja) 2022-07-28
JP2022534202A5 JP2022534202A5 (https=) 2023-05-31
JP7624410B2 true JP7624410B2 (ja) 2025-01-30

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US (1) US20220233654A1 (https=)
EP (1) EP3972631B1 (https=)
JP (1) JP7624410B2 (https=)
CN (1) CN114555813A (https=)
WO (1) WO2020234363A2 (https=)

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US20250262247A1 (en) * 2022-04-20 2025-08-21 The Regents Of The University Of California Microglial cells as therapeutic targets in neurodevelopmental disorders

Citations (5)

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WO2009034127A1 (en) 2007-09-12 2009-03-19 INSERM (Institut National de la Santé et de la Recherche Médicale) Use of the cyp46a1 gene for the treatment of alzheimer's disease
JP2013544236A (ja) 2010-10-15 2013-12-12 アンスティチュ ナショナル ドゥ ラ サンテ エ ドゥ ラ ルシェルシュ メディカル ハンチントン病の治療法におけるコレステロール−24−ヒドロラーゼのための発現ベクター
WO2018138371A1 (en) 2017-01-30 2018-08-02 Brainvectis Expression vector for cholesterol 24-hydrolase in therapy of polyglutamine repeat spinocerebellar ataxias
WO2018226785A1 (en) 2017-06-06 2018-12-13 University Of Massachusetts Self-regulating aav vectors for safe expression of mecp2 in rett syndrome
JP2019506141A (ja) 2015-12-11 2019-03-07 カリフォルニア インスティチュート オブ テクノロジー アデノ随伴ウイルス(aav)を誘導するための標的指向性ペプチド

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JP2019506141A (ja) 2015-12-11 2019-03-07 カリフォルニア インスティチュート オブ テクノロジー アデノ随伴ウイルス(aav)を誘導するための標的指向性ペプチド
WO2018138371A1 (en) 2017-01-30 2018-08-02 Brainvectis Expression vector for cholesterol 24-hydrolase in therapy of polyglutamine repeat spinocerebellar ataxias
WO2018226785A1 (en) 2017-06-06 2018-12-13 University Of Massachusetts Self-regulating aav vectors for safe expression of mecp2 in rett syndrome

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Publication number Publication date
WO2020234363A2 (en) 2020-11-26
WO2020234363A8 (en) 2021-10-07
CA3140809A1 (en) 2020-11-26
EP3972631A2 (en) 2022-03-30
US20220233654A1 (en) 2022-07-28
EP3972631B1 (en) 2024-07-10
CN114555813A (zh) 2022-05-27
WO2020234363A3 (en) 2021-11-18
JP2022534202A (ja) 2022-07-28
EP3972631C0 (en) 2024-07-10

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