| CPC C07K 14/4708 (2013.01) [A61K 48/0058 (2013.01); A61P 21/00 (2018.01); C07H 21/04 (2013.01); C07K 14/4707 (2013.01); C07K 14/4716 (2013.01); C12N 15/85 (2013.01); C12N 15/86 (2013.01); A61K 38/00 (2013.01); C07K 2319/30 (2013.01); C12N 2750/14143 (2013.01); C12N 2830/008 (2013.01)] | 6 Claims |
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1. A method for treating a subject having muscular dystrophy or prophylactically treating a subject at risk of developing muscular dystrophy, comprising:
administering to the subject a therapeutically effective amount of a pharmaceutical composition comprising a micro-dystrophin gene operably coupled to a regulatory cassette;
wherein the micro-dystrophin gene encodes a protein having at least 91% sequence identity to the amino acid sequence of SEQ ID NO: 4.
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