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.
Copyright Owner
Wiley Periodicals, Inc.
Copyright Date
2010
Language
en
File Format
application/pdf
Recommended Citation
Morine, Kevin J.; Bish, Lawrence T.; Selsby, Joshua T.; Gazzara, Jeffery A.; Pendrak, Klara; Sleeper, Meg M.; Barton, Elisabeth R.; Lee, Se-Jin; and Sweeney, H. Lee, "Activin IIB receptor blockade attenuates dystrophic pathology in a mouse model of duchenne muscular dystrophy" (2010). Animal Science Publications. 700.
https://lib.dr.iastate.edu/ans_pubs/700
Included in
Animal Experimentation and Research Commons, Animal Sciences Commons, Genetics and Genomics Commons, Musculoskeletal Diseases Commons
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.