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1. (WO2016115543) NOVEL MICRO-DYSTROPHINS AND RELATED METHODS OF USE

Pub. No.:    WO/2016/115543    International Application No.:    PCT/US2016/013733
Publication Date: Fri Jul 22 01:59:59 CEST 2016 International Filing Date: Sat Jan 16 00:59:59 CET 2016
IPC: C12N 15/12
C07K 14/47
A61K 48/00
Applicants: UNIVERSITY OF WASHINGTON
Inventors: CHAMBERLAIN, Jeffrey S.
RAMOS, Julian
HAUSCHKA, Stephen D.
Title: NOVEL MICRO-DYSTROPHINS AND RELATED METHODS OF USE
Abstract:
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.