Myostatin propeptide gene delivery by adeno-associated virus serotype 8 vectors enhances muscle growth and ameliorates dystrophic phenotypes in mdx mice.

Abstract excerpt

Myostatin has been extensively documented as a negative regulator of muscle growth. Myostatin inhibition is therefore considered an attractive strategy for the treatment of muscle-wasting diseases such as muscular dystrophies. To investigate whether systemic gene delivery of myostatin propeptide (MRPO), a natural inhibitor of myostatin, could enhance body-wide skeletal muscle growth, we used adeno-associated...

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