Key result
Systemic myostatin inhibition via liver-targeted gene transfer increased skeletal muscle mass in normal and dystrophic mice, but long-term treatment failed to rescue the diaphragm and induced cardiac hypertrophy.
Population
Normal C57 Bl/6 mice and dystrophin-deficient mdx mice (model of Duchenne muscular dystrophy)
Comparison
Intravenous injection of recombinant… vs Age-matched control mice
Design
Preclinical
Follow-up
up to 11 months
Authors
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Raises cardiac safety concerns with myostatin inhibition; leaves open long-term efficacy for diaphragm rescue in dystrophic models.
Liver-targeted gene transfer of a myostatin inhibitor increases skeletal muscle mass but causes long-term cardiac hypertrophy and impaired function in a mouse model of Duchenne muscular dystrophy.
Morine et al. (2010) studied Duchenne muscular dystrophy (mdx mouse model) (n=56). AAV2/8 LSP.dnMstat vs. Age-matched control mice was evaluated on Skeletal muscle mass and strength. Systemic myostatin inhibition via liver-targeted gene transfer increased skeletal muscle mass in normal and dystrophic mice, but long-term treatment failed to rescue the diaphragm and induced cardiac hypertrophy.
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