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BIOLOGICAL SCIENCES / MEDICAL SCIENCES
Long-term enhancement of skeletal muscle mass and strength by single gene administration of myostatin inhibitors
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*The Research Institute, Nationwide Children's Hospital, Columbus, OH 43205; and Integrated Biomedical Science and Neuroscience Graduate Programs, Ohio State University, Columbus, OH 43210
Edited by Joshua R. Sanes, Harvard University, Cambridge, MA, and approved February 5, 2008 (received for review September 25, 2007)
Increasing the size and strength of muscles represents a promising therapeutic strategy for musculoskeletal disorders, and interest has focused on myostatin, a negative regulator of muscle growth. Various myostatin inhibitor approaches have been identified and tested in models of muscle disease with varying efficacies, depending on the age at which myostatin inhibition occurs. Here, we describe a one-time gene administration of myostatin-inhibitor-proteins to enhance muscle mass and strength in normal and dystrophic mouse models for >2 years, even when delivered in aged animals. These results demonstrate a promising therapeutic strategy that warrants consideration for clinical trials in human muscle diseases.
gene therapy | muscular dystrophy
The authors declare no conflict of interest.
This article is a PNAS Direct Submission.
To whom correspondence should be addressed. E-mail: kasparb{at}ccri.net
© 2008 by The National Academy of Sciences of the USA
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