JP7507697B2 - 肢帯型筋ジストロフィー２ａを治療するための組換えアデノ随伴ウイルス産物及び方法 - Google Patents

肢帯型筋ジストロフィー２ａを治療するための組換えアデノ随伴ウイルス産物及び方法 Download PDF

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JP7507697B2
JP7507697B2 JP2020572953A JP2020572953A JP7507697B2 JP 7507697 B2 JP7507697 B2 JP 7507697B2 JP 2020572953 A JP2020572953 A JP 2020572953A JP 2020572953 A JP2020572953 A JP 2020572953A JP 7507697 B2 JP7507697 B2 JP 7507697B2
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ザリフェ サヘンク，
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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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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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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
    • 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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • A—HUMAN NECESSITIES
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
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    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/48—Hydrolases (3) acting on peptide bonds (3.4)
    • C12N9/50—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
    • C12N9/64—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
    • C12N9/6421—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue from mammals
    • C12N9/6472—Cysteine endopeptidases (3.4.22)
    • C—CHEMISTRY; METALLURGY
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14131—Uses of virus other than therapeutic or vaccine, e.g. disinfectant
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • 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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  • Chemical Kinetics & Catalysis (AREA)
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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
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JP2020572953A 2018-06-29 2019-06-28 肢帯型筋ジストロフィー２ａを治療するための組換えアデノ随伴ウイルス産物及び方法 Active JP7507697B2 (ja)

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JP2024098061A JP2024123110A (ja) 2018-06-29 2024-06-18 肢帯型筋ジストロフィー２ａを治療するための組換えアデノ随伴ウイルス産物及び方法

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US201862691934P 2018-06-29 2018-06-29
US62/691,934 2018-06-29
US201962865081P 2019-06-21 2019-06-21
US62/865,081 2019-06-21
PCT/US2019/039893 WO2020006458A1 (en) 2018-06-29 2019-06-28 Recombinant adeno-associated virus products and methods for treating limb girdle muscular dystrophy 2a

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JP2024098061A Pending JP2024123110A (ja) 2018-06-29 2024-06-18 肢帯型筋ジストロフィー２ａを治療するための組換えアデノ随伴ウイルス産物及び方法

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KR (1) KR20210028162A (enExample)
CN (1) CN112543810A (enExample)
AU (2) AU2019295779B2 (enExample)
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US11338045B2 (en) 2017-03-17 2022-05-24 Newcastle University Adeno-associated virus vector delivery of a fragment of micro-dystrophin to treat muscular dystrophy
AU2019216257B2 (en) 2018-01-31 2025-11-20 Research Institute At Nationwide Children's Hospital Gene therapy for limb-girdle muscular dystrophy type 2C
CN120393053A (zh) 2018-06-18 2025-08-01 全国儿童医院研究所 用于治疗肌营养不良的肌肉特异性微小肌营养不良蛋白的腺相关病毒载体递送
US12377170B2 (en) 2019-02-26 2025-08-05 Research Institute At Nationwide Children's Hospital Adeno-associated virus vector delivery of β-sarcoglycan and the treatment of muscular dystrophy
EP4017871B1 (en) 2019-08-21 2024-01-17 Research Institute at Nationwide Children's Hospital Adeno-associated virus vector delivery of alpha-sarcoglycan and the treatment of muscular dystrophy
IL299094A (en) 2020-06-15 2023-02-01 Res Inst Nationwide Childrens Hospital Administration of an adeno-associated virus vector for muscular dystrophies
CN116194587A (zh) * 2020-07-10 2023-05-30 吉尼松公司 一种新的肌肉特异性启动子
EP4108263A3 (en) 2021-06-02 2023-03-22 Research Institute at Nationwide Children's Hospital Recombinant adeno-associated virus products and methods for treating limb girdle muscular dystrophy 2a
USD1008419S1 (en) * 2021-06-14 2023-12-19 Jong Won Kim Vent valve of a wastewater drainage pipe
JP2023059858A (ja) 2021-10-15 2023-04-27 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 自己相補的アデノ随伴ウイルスベクター及び筋ジストロフィーの治療におけるその使用
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WO2024216116A1 (en) * 2023-04-14 2024-10-17 Precision Biosciences, Inc. Muscle-specific expression cassettes
WO2025226842A1 (en) 2024-04-24 2025-10-30 Kate Therapeutics, Inc. Expression control by drg-expressed mirnas

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