Targeting Myostatin for Therapies Against Muscle-Wasting Disorders

Curr Opin Drug Discov Devel. 2008 Jul;11(4):487-94.

Abstract

In addition to gene correction therapy and cell transplantation techniques, multidisciplinary approaches to drug discovery and development offer promising therapeutic strategies for intractable genetic muscular disorders including muscular dystrophy. Inhibition of the production and activity of myostatin, a potent growth factor that determines skeletal muscle size, is a novel strategy for the treatment of muscle-wasting disorders such as muscular dystrophy, cachexia and sarcopenia. Myostatin blockers include myostatin-blocking antibodies, myostatin propeptide, follistatin and follistatin-related proteins, soluble myostatin receptors, small interfering RNA and small chemical inhibitors. This review describes the discovery and development of myostatin inhibitors.

Publication types

  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Amino Acid Sequence
  • Animals
  • Antibodies / pharmacology
  • Antibodies / therapeutic use
  • Drug Design*
  • Follistatin / pharmacology
  • Follistatin / therapeutic use
  • Genetic Therapy / methods
  • Humans
  • Models, Molecular
  • Molecular Sequence Data
  • Muscle, Skeletal / drug effects*
  • Muscle, Skeletal / metabolism
  • Muscular Atrophy / drug therapy
  • Muscular Atrophy / genetics
  • Muscular Atrophy / metabolism
  • Muscular Atrophy / therapy*
  • Myostatin
  • Peptides / pharmacology
  • Peptides / therapeutic use
  • Protein Conformation
  • RNA Interference
  • Signal Transduction / drug effects
  • Transforming Growth Factor beta / antagonists & inhibitors*
  • Transforming Growth Factor beta / chemistry
  • Transforming Growth Factor beta / genetics
  • Transforming Growth Factor beta / metabolism

Substances

  • Antibodies
  • Follistatin
  • MSTN protein, human
  • Myostatin
  • Peptides
  • Transforming Growth Factor beta