Muscle regeneration through myostatin inhibition.
OBJECTIVE
Myostatin is an endogenous, negative regulator of muscle growth. Selective inhibition of myostatin may have broad clinical utility by improving regeneration in diverse and burdensome muscle disorders. An understanding of this potential is relevant because inhibitors of myostatin have recently entered clinical trials.


RESULTS
This article reviews the structure and function of myostatin, the effect of inhibiting myostatin in models of disease, and potential therapeutic approaches to blocking myostatin pharmacologically. The possibility that a myostatin inhibitor will promote muscle regeneration in human disease, as seen in animal models, is suggested by the observation that loss of myostatin results in muscle hypertrophy in a human subject.


CONCLUSIONS
Multiple approaches to inhibiting myostatin are suggested by the recent elucidation of its signaling pathway. An inhibitor of myostatin may be the first drug specifically designed to enhance muscle growth and regeneration.
Wagner
Kathryn R
KR
Department of Neurology, Johns Hopkins School of Medicine, Baltimore, Maryland 21287, USA. kwagner@jhmi.edu
D000818:Animals; D002454:Cell Differentiation / Q000187:drug effects; D038681:Follistatin / Q000627:therapeutic use*; D038702:Follistatin-Related Proteins / Q000627:therapeutic use*; D006801:Humans; D018482:Muscle, Skeletal / Q000502:physiology*; D009135:Muscular Diseases / Q000378:metabolism / Q000473:pathology / Q000517:prevention & control; D055435:Myostatin; D012038:Regeneration / Q000502:physiology*; D016212:Transforming Growth Factor beta / Q000037:antagonists & inhibitors*; D033921:Vesicular Transport Proteins / Q000627:therapeutic use*
D038681:Follistatin; D038702:Follistatin-Related Proteins; C463459:GPRASP1 protein, human; C525911:MSTN protein, human; D055435:Myostatin; D016212:Transforming Growth Factor beta; D033921:Vesicular Transport Proteins