Campus Units

Animal Science

Document Type

Article

Publication Version

Accepted Manuscript

Publication Date

11-2010

Journal or Book Title

Muscle & Nerve

Volume

42

Issue

5

First Page

722

Last Page

730

DOI

10.1002/mus.21743

Abstract

Modulation of transforming growth factor-β (TGF-β) signaling to promote muscle growth holds tremendous promise for the muscular dystrophies and other disorders involving the loss of functional muscle mass. Previous studies have focused on the TGF-β family member myostatin and demonstrated that inhibition of myostatin leads to muscle growth in normal and dystrophic mice. We describe a unique method of systemic inhibition of activin IIB receptor signaling via adeno-associated virus (AAV)-mediated gene transfer of a soluble form of the extracellular domain of the activin IIB receptor to the liver. Treatment of mdx mice with activin IIB receptor blockade led to increased skeletal muscle mass, increased force production in the extensor digitorum longus (EDL), and reduced serum creatine kinase. No effect on heart mass or function was observed. Our results indicate that activin IIB receptor blockade represents a novel and effective therapeutic strategy for the muscular dystrophies.

Comments

This is the peer reviewed version of the following article: Morine, Kevin J., Lawrence T. Bish, Joshua T. Selsby, Jeffery A. Gazzara, Klara Pendrak, Meg M. Sleeper, Elisabeth R. Barton, Se‐Jin Lee, and H. Lee Sweeney. "Activin IIB receptor blockade attenuates dystrophic pathology in a mouse model of Duchenne muscular dystrophy." Muscle & nerve 42, no. 5 (2010): 722-730, which has been published in final form at doi: 10.1002/mus.21743. This article may be used for non-commercial purposes in accordance with Wiley Terms and Conditions for Use of Self-Archived Versions.

Copyright Owner

Wiley Periodicals, Inc.

Language

en

File Format

application/pdf

Published Version

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