Actriib proteins and variants and uses therefore relating to utrophin induction for muscular dystrophy therapy
AssigneeAcceleron Pharma Inc.
InventorsJasbir Seehra, Jennifer Lachey
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Abstract
In certain aspects, the present invention provides compositions and methods for inducing utrophin expression in muscle with an ActRIIB protein as therapy for muscular dystrophy. The present invention also provides methods of screening compounds that modulate activity of an ActRIIB protein and/or an ActRIIB ligand.
Claims20
1-18. (canceled)
A method for increasing sarcolemmal utrophin in a patient in need thereof, the method comprising administering an effective amount of an antibody or antigen-binding fragment thereof that binds to ActRIIB.
20-27. (canceled)
The method of claim 1, wherein the method further comprises evaluating a marker for muscle degeneration and selecting a dose level or frequency based on the level of the marker for muscle degeneration.
34-49. (canceled)
A method for increasing sarcolemmal utrophin in a patient in need thereof, the method comprising administering an effective amount of an antisense or RNAi nucleic acid that inhibits the expression of ActRIIB.
Dependencies are inferred from the claim text.