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.
Claims30
A method for increasing sarcolemmal utrophin in a patient in need thereof, the method comprising administering to the patient an effective amount of a polypeptide selected from the group consisting of:
- a. a polypeptide comprising the amino acid sequence of SEQ ID NO:5 and
- b. a polypeptide comprising the amino acid sequence of SEQ ID NO:23.
A method for treating a DMD or BMD patient that has an elevated marker for muscle degeneration, the method comprising administering to the patient an effective amount of a polypeptide selected from the group consisting of:
- a. a polypeptide comprising the amino acid sequence of SEQ ID NO:5;
- and
- b. a polypeptide comprising the amino acid sequence of SEQ ID NO:23.
A method for treating DMD or BMD in a patient in need thereof, the method comprising administering to the patient an effective amount of a polypeptide:
- comprising the amino acid sequence of SEQ ID NO:23.
Dependencies are inferred from the claim text.