JP7575273B2 - デュシェンヌ型筋ジストロフィーを治療するための組成物及び方法 - Google Patents

デュシェンヌ型筋ジストロフィーを治療するための組成物及び方法 Download PDF

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JP7575273B2
JP7575273B2 JP2020557155A JP2020557155A JP7575273B2 JP 7575273 B2 JP7575273 B2 JP 7575273B2 JP 2020557155 A JP2020557155 A JP 2020557155A JP 2020557155 A JP2020557155 A JP 2020557155A JP 7575273 B2 JP7575273 B2 JP 7575273B2
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ステッドマン，ハンセル
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ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア
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    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4707—Muscular dystrophy
    • C07K14/4708—Duchenne dystrophy
    • C—CHEMISTRY; METALLURGY
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    • A01K2227/00—Animals characterised by species
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    • A01K2227/105—Murine
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • A01K2267/00—Animals characterised by purpose
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14121—Viruses as such, e.g. new isolates, mutants or their genomic sequences
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
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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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  • Health & Medical Sciences (AREA)
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JP2020557155A 2018-04-16 2019-04-16 デュシェンヌ型筋ジストロフィーを治療するための組成物及び方法 Active JP7575273B2 (ja)

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US201862658464P 2018-04-16 2018-04-16
US62/658,464 2018-04-16
PCT/US2019/027675 WO2019204303A2 (en) 2018-04-16 2019-04-16 Compositions and methods for treating duchenne muscular dystrophy

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JP2021521782A JP2021521782A (ja) 2021-08-30
JP2021521782A5 JP2021521782A5 (https=) 2022-04-19
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EP3813871A4 (en) * 2018-06-29 2022-10-12 Icahn School of Medicine at Mount Sinai SPHINGOLIPID METABOLIZING PROTEINS ENCODING FOR ANC80
IL294713A (en) * 2020-01-16 2022-09-01 Takeda Pharmaceuticals Co Adeno associated virus based gene therapy for phenylketonuria
CN113999861B (zh) * 2021-09-08 2024-03-15 三峡大学 腺相关病毒衣壳蛋白保守区多克隆抗体的制备方法及应用

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BR112020021179A2 (pt) 2021-03-02
CA3099958A1 (en) 2019-10-24
US20210155956A1 (en) 2021-05-27
CN112601454B (zh) 2023-10-27
WO2019204303A2 (en) 2019-10-24
EP3780952A2 (en) 2021-02-24
WO2019204303A3 (en) 2019-11-28
JP2021521782A (ja) 2021-08-30
US20260109998A1 (en) 2026-04-23
US12460226B2 (en) 2025-11-04
EP3780952A4 (en) 2022-03-09
CN112601454A (zh) 2021-04-02

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