TWI854984B - 用於治療肢帶型肌營養不良症2a之重組腺相關病毒產品與方法 - Google Patents

用於治療肢帶型肌營養不良症2a之重組腺相關病毒產品與方法 Download PDF

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TWI854984B
TWI854984B TW108123006A TW108123006A TWI854984B TW I854984 B TWI854984 B TW I854984B TW 108123006 A TW108123006 A TW 108123006A TW 108123006 A TW108123006 A TW 108123006A TW I854984 B TWI854984 B TW I854984B
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札瑞芙 撒漢克
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美國全美兒童醫院之研究學會
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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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    • 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
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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
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    • 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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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • Chemical Kinetics & Catalysis (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Immunology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Peptides Or Proteins (AREA)
TW108123006A 2018-06-29 2019-06-28 用於治療肢帶型肌營養不良症2a之重組腺相關病毒產品與方法 TWI854984B (zh)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
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

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TW202020161A TW202020161A (zh) 2020-06-01
TWI854984B true TWI854984B (zh) 2024-09-11

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US (1) US12391928B2 (enExample)
EP (1) EP3814514A1 (enExample)
JP (2) JP7507697B2 (enExample)
KR (1) KR20210028162A (enExample)
CN (1) CN112543810A (enExample)
AU (2) AU2019295779B2 (enExample)
BR (1) BR112020026853A2 (enExample)
CA (1) CA3105216A1 (enExample)
CL (1) CL2020003384A1 (enExample)
CO (1) CO2021000914A2 (enExample)
IL (1) IL279789B1 (enExample)
MX (2) MX2020014119A (enExample)
SG (1) SG11202012715UA (enExample)
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WO (1) WO2020006458A1 (enExample)

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HK1257955A1 (zh) 2015-11-16 2019-11-01 The Research Institute At Nationwide Children's Hospital 用於治疗肌联蛋白类肌病和其他肌联蛋白病变的材料和方法
JP7162021B2 (ja) 2017-03-17 2022-10-27 ニューカッスル ユニバーシティ 筋ジストロフィーを治療するためのマイクロジストロフィン断片のアデノ随伴ウイルスベクター送達
JP7361701B2 (ja) 2018-01-31 2023-10-16 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 肢帯型筋ジストロフィー２ｃ型のための遺伝子治療
JP7646362B2 (ja) 2018-06-18 2025-03-17 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 筋ジストロフィーを治療するための筋肉特異的マイクロジストロフィンのアデノ随伴ウイルスベクターデリバリー
EP3930765A1 (en) 2019-02-26 2022-01-05 Research Institute at Nationwide Children's Hospital Adeno-associated virus vector delivery of b-sarcoglycan and the treatment of muscular dystrophy
SI4017871T1 (sl) 2019-08-21 2024-06-28 Research Institute At Nationwide Children's Hospital Dostava alfa-sarkoglikana z adeno povezanim virusnim vektorjem in zdravljenje mišične distrofije
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