JP7534961B2 - ジストログリカノパチーおよびラミニン欠損筋ジストロフィーを治療するための組換えアデノ随伴ウイルス生成物および方法 - Google Patents

ジストログリカノパチーおよびラミニン欠損筋ジストロフィーを治療するための組換えアデノ随伴ウイルス生成物および方法 Download PDF

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JP7534961B2
JP7534961B2 JP2020570457A JP2020570457A JP7534961B2 JP 7534961 B2 JP7534961 B2 JP 7534961B2 JP 2020570457 A JP2020570457 A JP 2020570457A JP 2020570457 A JP2020570457 A JP 2020570457A JP 7534961 B2 JP7534961 B2 JP 7534961B2
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ポール テイラー マーティン,
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リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル
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    • C07K14/475Growth factors; Growth regulators
    • C07K14/485Epidermal growth factor [EGF], i.e. urogastrone
    • AHUMAN NECESSITIES
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    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
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    • C07K14/78Connective tissue peptides, e.g. collagen, elastin, laminin, fibronectin, vitronectin or cold insoluble globulin [CIG]
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    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
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    • C12N15/86Viral vectors
    • AHUMAN NECESSITIES
    • 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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    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/008Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination

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  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
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  • Plant Pathology (AREA)
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  • Virology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
JP2020570457A 2018-06-18 2019-06-18 ジストログリカノパチーおよびラミニン欠損筋ジストロフィーを治療するための組換えアデノ随伴ウイルス生成物および方法 Active JP7534961B2 (ja)

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JP2024127744A JP2024156872A (ja) 2018-06-18 2024-08-02 ジストログリカノパチーおよびラミニン欠損筋ジストロフィーを治療するための組換えアデノ随伴ウイルス生成物および方法

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US201862686522P 2018-06-18 2018-06-18
US62/686,522 2018-06-18
PCT/US2019/037769 WO2019246125A1 (en) 2018-06-18 2019-06-18 Recombinant adeno-associated virus products and methods for treating dystroglycanopathies and laminin-deficient muscular dystrophies

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JP2024127744A Pending JP2024156872A (ja) 2018-06-18 2024-08-02 ジストログリカノパチーおよびラミニン欠損筋ジストロフィーを治療するための組換えアデノ随伴ウイルス生成物および方法

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US (1) US20240254181A1 (https=)
EP (1) EP3807309A1 (https=)
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AU (2) AU2019288291B2 (https=)
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WO2017181015A1 (en) 2016-04-15 2017-10-19 Research Institute At Nationwide Children's Hospital Adeno-associated virus vector delivery of micro-dystrophin to treat muscular dystrophy

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PT728214E (pt) 1993-11-09 2004-11-30 Ohio Med College Linhas celulares estaveis capazes de expressar o gene de replicacao do virus adeno-associado
PT733103E (pt) 1993-11-09 2004-07-30 Targeted Genetics Corp Criacao de elevados titulos de vectores de aav recombinantes
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AU2019288291A1 (en) 2021-01-21
US20240254181A1 (en) 2024-08-01
AU2019288291B2 (en) 2024-09-26
EP3807309A1 (en) 2021-04-21
AU2024259685A1 (en) 2024-12-05
CA3104471A1 (en) 2019-12-26
JP2021527418A (ja) 2021-10-14
WO2019246125A1 (en) 2019-12-26
WO2019246125A8 (en) 2020-05-14
JP2024156872A (ja) 2024-11-06

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