Adeno-associated virus-8-mediated intravenous transfer of myostatin propeptide leads to systemic functional improvements of slow but not fast muscle

Rejuvenation Res. 2009 Apr;12(2):85-94. doi: 10.1089/rej.2008.0815.

Abstract

Myostatin is a member of the transformating growth factor-beta (TGF-beta) superfamily of proteins and is produced almost exclusively in skeletal muscle tissue, where it is secreted and circulates as a serum protein. Myostatin acts as a negative regulator of muscle mass through the canonical SMAD2/3/4 signaling pathway. Naturally occurring myostatin mutants exhibit a 'double muscling' phenotype in which muscle mass is dramatically increased as a result of both hypertrophy and hyperplasia. Myostatin is naturally inhibited by its own propeptide; therefore, we assessed the impact of adeno-associated virus-8 (AAV8) myostatin propeptide vectors when systemically introduced in MF-1 mice. We noted a significant systemic increase in muscle mass in both slow and fast muscle phenotypes, with no evidence of hyperplasia; however, the nuclei-to- cytoplasm ratio in all myofiber types was significantly reduced. An increase in muscle mass in slow (soleus) muscle led to an increase in force output; however, an increase in fast (extensor digitorum longus [EDL]) muscle mass did not increase force output. These results suggest that the use of gene therapeutic regimens of myostatin inhibition for age-related or disease-related muscle loss may have muscle-specific effects.

MeSH terms

  • Animals
  • Biomechanical Phenomena / drug effects
  • Body Weight / drug effects
  • Cell Nucleus / drug effects
  • Cell Nucleus / metabolism
  • Dependovirus / metabolism*
  • Gene Transfer Techniques*
  • Hyperplasia
  • Hypertrophy
  • Injections, Intravenous
  • Mice
  • Muscle Fibers, Fast-Twitch / drug effects
  • Muscle Fibers, Fast-Twitch / pathology
  • Muscle Fibers, Fast-Twitch / physiology*
  • Muscle Fibers, Slow-Twitch / drug effects
  • Muscle Fibers, Slow-Twitch / pathology
  • Muscle Fibers, Slow-Twitch / physiology*
  • Myostatin / administration & dosage*
  • Myostatin / antagonists & inhibitors
  • Myostatin / metabolism
  • Myostatin / pharmacology
  • Organ Size / drug effects
  • Peptides / administration & dosage*
  • Peptides / metabolism
  • Peptides / pharmacology
  • Protein Precursors / administration & dosage*
  • Protein Precursors / metabolism
  • Protein Precursors / pharmacology
  • Satellite Cells, Skeletal Muscle / drug effects
  • Satellite Cells, Skeletal Muscle / pathology

Substances

  • Myostatin
  • Peptides
  • Protein Precursors