US 12,421,285 B2
Micro-dystrophins and related methods of use
Jeffrey S. Chamberlain, Seattle, WA (US); Julian Ramos, Seattle, WA (US); and Stephen D. Hauschka, Seattle, WA (US)
Assigned to University of Washington, Seattle, WA (US)
Filed by University of Washington, Seattle, WA (US)
Filed on Oct. 4, 2019, as Appl. No. 16/593,840.
Application 16/593,840 is a division of application No. 15/541,870, granted, now 10,479,821, previously published as PCT/US2016/013733, filed on Jan. 15, 2016.
Claims priority of provisional application 62/104,537, filed on Jan. 16, 2015.
Prior Publication US 2020/0031890 A1, Jan. 30, 2020
Int. Cl. C07K 14/47 (2006.01); A61K 38/00 (2006.01); A61K 48/00 (2006.01); A61P 21/00 (2006.01); C07H 21/04 (2006.01); C12N 15/85 (2006.01); C12N 15/86 (2006.01)
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
 
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.