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公开(公告)号:EP4319874A2
公开(公告)日:2024-02-14
申请号:EP22785484.1
申请日:2022-04-07
IPC分类号: A61P21/00 , C12N15/66 , C12N15/67 , C12N15/85 , C12N15/861
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公开(公告)号:EP3245291A2
公开(公告)日:2017-11-22
申请号:EP16738022.9
申请日:2016-01-15
CPC分类号: C07K14/4708 , A61K38/00 , A61K48/0058 , A61P21/00 , C07H21/04 , C07K14/4707 , C07K14/4716 , C07K2319/30 , C12N15/85 , C12N2750/14143 , C12N2830/008
摘要: Nucleotide sequences including a micro-dystrophin gene are provided. The micro-dystrophin genes may be operatively linked to a regulatory cassette. Methods of treating a subject having, or at risk of developing, muscular dystrophy, sarcopenia, heart disease, or cachexia are also provided. The methods may include administering a pharmaceutical composition including the micro-dystrophin gene and a delivery vehicle to a subject. Further, the methods may include administering the pharmaceutical composition a subject having Duchenne muscular dystrophy or Becker muscular dystrophy.
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