Loss of myostatin attenuates severity of muscular dystrophy in mdx mice

Ann Neurol. 2002 Dec;52(6):832-6. doi: 10.1002/ana.10385.


Myostatin, a transforming growth factor-beta family member, is a negative regulator of skeletal muscle growth. To explore the therapeutic potential of targeting myostatin in settings of muscle degeneration, we crossed myostatin null mutant mice with mdx mice, a model for Duchenne and Becker muscular dystrophy. Mdx mice lacking myostatin were stronger and more muscular than their mdx counterparts. Diaphragm muscle showed less fibrosis and fatty remodeling, suggesting improved muscle regeneration.

Publication types

  • Comparative Study
  • Research Support, Non-U.S. Gov't
  • Research Support, U.S. Gov't, P.H.S.

MeSH terms

  • Animals
  • Female
  • Male
  • Mice
  • Mice, Inbred C57BL
  • Mice, Inbred mdx
  • Mice, Mutant Strains
  • Muscle Fibers, Skeletal / pathology
  • Muscular Dystrophies / genetics*
  • Muscular Dystrophies / pathology*
  • Muscular Dystrophies / physiopathology
  • Myostatin
  • Severity of Illness Index
  • Transforming Growth Factor beta / deficiency*
  • Transforming Growth Factor beta / genetics*


  • Mstn protein, mouse
  • Myostatin
  • Transforming Growth Factor beta