Patent ID: 11958888
Assignee: UNIVERSITY OF WASHINGTON
Field: Pharmaceuticals (Chemistry)
Classification: CPC C  A | IPC A  C

Claim 0:
1. A pharmaceutical composition, the composition comprising:
a micro-dystrophin gene encoding a protein comprising:
an amino-terminal actin-binding domain;
a β-dystroglycan binding domain; and
a spectrin-like repeat domain consisting of five spectrin-like repeats, including spectrin-like repeat 1 (SR1), spectrin-like repeat 16 (SR16), spectrin-like repeat 17 (SR17), spectrin-like repeat 23 (SR23), and spectrin-like repeat 24 (SR24);, wherein the micro-dystrophin gene is operatively linked to a regulatory cassette; and
a delivery vehicle, wherein the pharmaceutical composition is formulated for intravenous or intramuscular administration.