JP2019513399A5 - - Google Patents

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JP2019513399A5
JP2019513399A5 JP2018554055A JP2018554055A JP2019513399A5 JP 2019513399 A5 JP2019513399 A5 JP 2019513399A5 JP 2018554055 A JP2018554055 A JP 2018554055A JP 2018554055 A JP2018554055 A JP 2018554055A JP 2019513399 A5 JP2019513399 A5 JP 2019513399A5
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JP
Japan
Prior art keywords
aav vector
recombinant aav
composition
seq
muscle
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JP2018554055A
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English (en)
Japanese (ja)
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JP7079733B2 (ja
JP2019513399A (ja
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Priority claimed from PCT/US2017/027583 external-priority patent/WO2017180976A1/en
Publication of JP2019513399A publication Critical patent/JP2019513399A/ja
Publication of JP2019513399A5 publication Critical patent/JP2019513399A5/ja
Priority to JP2022061771A priority Critical patent/JP7390426B2/ja
Application granted granted Critical
Publication of JP7079733B2 publication Critical patent/JP7079733B2/ja
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JP2018554055A 2016-04-15 2017-04-14 β-サルコグリカン及びマイクロRNA-29のアデノ随伴ウイルスベクター送達ならびに筋ジストロフィーの治療 Active JP7079733B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2022061771A JP7390426B2 (ja) 2016-04-15 2022-04-01 β-サルコグリカン及びマイクロRNA-29のアデノ随伴ウイルスベクター送達ならびに筋ジストロフィーの治療

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201662323333P 2016-04-15 2016-04-15
US62/323,333 2016-04-15
US201662433548P 2016-12-13 2016-12-13
US62/433,548 2016-12-13
PCT/US2017/027583 WO2017180976A1 (en) 2016-04-15 2017-04-14 Adeno-associated virus vector delivery of b-sarcoglycan and microrna-29 and the treatment of muscular dystrophy

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2022061771A Division JP7390426B2 (ja) 2016-04-15 2022-04-01 β-サルコグリカン及びマイクロRNA-29のアデノ随伴ウイルスベクター送達ならびに筋ジストロフィーの治療

Publications (3)

Publication Number Publication Date
JP2019513399A JP2019513399A (ja) 2019-05-30
JP2019513399A5 true JP2019513399A5 (enExample) 2020-05-14
JP7079733B2 JP7079733B2 (ja) 2022-06-02

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ID=60042261

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2018554055A Active JP7079733B2 (ja) 2016-04-15 2017-04-14 β-サルコグリカン及びマイクロRNA-29のアデノ随伴ウイルスベクター送達ならびに筋ジストロフィーの治療
JP2022061771A Active JP7390426B2 (ja) 2016-04-15 2022-04-01 β-サルコグリカン及びマイクロRNA-29のアデノ随伴ウイルスベクター送達ならびに筋ジストロフィーの治療

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JP2022061771A Active JP7390426B2 (ja) 2016-04-15 2022-04-01 β-サルコグリカン及びマイクロRNA-29のアデノ随伴ウイルスベクター送達ならびに筋ジストロフィーの治療

Country Status (28)

Country Link
US (3) US11358993B2 (enExample)
EP (2) EP3442600B1 (enExample)
JP (2) JP7079733B2 (enExample)
KR (1) KR102502091B1 (enExample)
CN (2) CN109121395B (enExample)
AU (2) AU2017250756B2 (enExample)
BR (1) BR112018071167A2 (enExample)
CA (1) CA3021057A1 (enExample)
CO (1) CO2018012082A2 (enExample)
DK (1) DK3442600T5 (enExample)
ES (1) ES2979174T3 (enExample)
FI (1) FI3442600T3 (enExample)
HR (1) HRP20240580T1 (enExample)
HU (1) HUE066442T2 (enExample)
IL (1) IL262262B2 (enExample)
LT (1) LT3442600T (enExample)
MA (1) MA44681A (enExample)
MX (2) MX391208B (enExample)
MY (1) MY195439A (enExample)
PL (1) PL3442600T3 (enExample)
PT (1) PT3442600T (enExample)
RS (1) RS65490B1 (enExample)
SA (1) SA518400245B1 (enExample)
SG (1) SG11201809029RA (enExample)
SI (1) SI3442600T1 (enExample)
SM (1) SMT202400169T1 (enExample)
WO (1) WO2017180976A1 (enExample)
ZA (1) ZA201806863B (enExample)

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JP2022523038A (ja) * 2019-02-02 2022-04-21 ユニバーシティ オブ ピッツバーグ -オブ ザ コモンウェルス システム オブ ハイヤー エデュケイション 遺伝子置換及び抗炎症を介したデュシェンヌ型筋ジストロフィーに対する一段階遺伝子療法
JP2022521776A (ja) * 2019-02-26 2022-04-12 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 Β-サルコグリカンのアデノ随伴ウイルスベクター送達および筋ジストロフィーの治療
MY210230A (en) * 2019-08-21 2025-09-04 Res Inst Nationwide Childrens Hospital Adeno-associated virus vector delivery of alpha-sarcoglycan and the treatment of muscular dystrophy
EP4117783A4 (en) * 2019-12-16 2024-08-21 Research Institute at Nationwide Children's Hospital COMPOSITIONS AND METHODS FOR RESTORING AND MAINTAINING DYSTROPHIN-ASSOCIATED PROTEIN COMPLEX (DAPC)
CA3182313A1 (en) * 2020-06-19 2021-12-23 Isabelle Richard Gene therapy expression system allowing an adequate expression in the muscles and in the heart of sgcg
EP4219726A1 (en) 2021-10-15 2023-08-02 Research Institute at Nationwide Children's Hospital Self-complementary adeno-associated virus vector and its use in treatment of muscular dystrophy
CN119731321A (zh) 2022-06-24 2025-03-28 图恩疗法股份有限公司 通过靶向基因阻遏减少低密度脂蛋白的组合物、系统和方法
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CN118045206B (zh) * 2024-04-12 2024-07-05 四川至善唯新生物科技有限公司 一种治疗脊髓型肌肉萎缩的药物组合物及其用途

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