Role of TGF-β signaling in inherited and acquired myopathies

Tyesha N Burks1, Ronald D Cohn

  • 1McKusick-Nathans Institute of Genetic Medicine, Johns Hopkins University School of Medicine, Baltimore, MD 21205, USA.

Skeletal Muscle
|July 30, 2011
PubMed

Insights

Transforming growth factor-beta (TGF-β) signaling is vital for skeletal muscle but aberrant TGF-β1, MAPKs, and myostatin contribute to muscle disorders. Inhibiting this pathway offers therapeutic potential for neuromuscular diseases.

Area of Science:

  • Molecular Biology
  • Cell Biology
  • Physiology

Background:

  • The transforming growth factor-beta (TGF-β) superfamily, including TGF-β1, MAPKs, and myostatin, is crucial for skeletal muscle function.
  • These molecules regulate muscle growth, differentiation, regeneration, and stress responses in skeletal muscle homeostasis.

Purpose of the Study:

  • To review the role of aberrant TGF-β signaling in various neuromuscular disorders.
  • To explore the therapeutic potential of inhibiting TGF-β pathway members for treating muscle pathologies.

Main Methods:

  • Literature review focusing on TGF-β superfamily members in skeletal muscle.
  • Analysis of signaling pathways implicated in muscle homeostasis and disease.
  • Examination of therapeutic strategies targeting TGF-β signaling.

Main Results:

  • Chronic elevation of TGF-β1, MAPKs, and myostatin is associated with impaired muscle regeneration and atrophy.
  • Aberrant TGF-β signaling is linked to conditions like Marfan syndrome, muscular dystrophies, sarcopenia, and critical illness myopathy.

Conclusions:

  • Dysregulated TGF-β signaling significantly contributes to the pathogenesis of numerous neuromuscular disorders.
  • Targeting the TGF-β pathway presents promising therapeutic avenues for ameliorating muscle disease phenotypes.

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