KR20210028162A - 지대근 이영양증 2a형을 치료하기 위한 재조합 아데노 연관 바이러스 생성물 및 방법 - Google Patents

지대근 이영양증 2a형을 치료하기 위한 재조합 아데노 연관 바이러스 생성물 및 방법 Download PDF

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KR20210028162A
KR20210028162A KR1020207037223A KR20207037223A KR20210028162A KR 20210028162 A KR20210028162 A KR 20210028162A KR 1020207037223 A KR1020207037223 A KR 1020207037223A KR 20207037223 A KR20207037223 A KR 20207037223A KR 20210028162 A KR20210028162 A KR 20210028162A
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muscle
raav
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자리프 사헨크
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더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈
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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
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    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
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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
    • C12N9/6472—Cysteine endopeptidases (3.4.22)
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    • 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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    • 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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  • 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)
KR1020207037223A 2018-06-29 2019-06-28 지대근 이영양증 2a형을 치료하기 위한 재조합 아데노 연관 바이러스 생성물 및 방법 Pending KR20210028162A (ko)

Applications Claiming Priority (5)

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
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

Publications (1)

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KR20210028162A true KR20210028162A (ko) 2021-03-11

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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)
TW (1) TWI854984B (enExample)
WO (1) WO2020006458A1 (enExample)

Cited By (1)

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USD1008419S1 (en) * 2021-06-14 2023-12-19 Jong Won Kim Vent valve of a wastewater drainage pipe

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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
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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
WO2021257595A1 (en) 2020-06-15 2021-12-23 Research Institute At Nationwide Children's Hospital Adeno-associated virus vector delivery for muscular dystrophies
CA3184923A1 (en) * 2020-07-10 2022-01-13 William LOSTAL A novel muscle-specific promoter
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
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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