Micro-dystrophins and related methods of use
AssigneeUniversity of Washington
InventorsJeffrey S. Chamberlain, Julian Ramos, Stephen D. Hauschka
Life of this patent
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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.
Claims16
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.
Dependencies are inferred from the claim text.