Targeting fibrosis in duchenne muscular dystrophy

Lan Zhou, Haiyan Lu

Research output: Contribution to journalReview article

84 Citations (Scopus)

Abstract

Duchenne muscular dystrophy (DMD) is the most common genetic muscle disease affecting 1 in 3,500 live male births. It is an X-linked recessive disease caused by a defective dystrophin gene. The disease is characterized by progressive limb weakness, respiratory and cardiac failure, and premature death. Fibrosis is a prominent pathological feature of muscle biopsies from patients with DMD. It directly causes muscle dysfunction and contributes to the lethal DMD phenotype. Although gene therapy and cell therapy may ultimately provide a cure for DMD, currently the disease is devastating, with no effective therapies. Recent studies have demonstrated that ameliorating muscle fibrosis may represent a viable therapeutic approach for DMD. By reducing scar formation, antifibrotic therapies may not only improve muscle function but also enhance muscle regeneration and promote gene and stem cell engraftment. Antifibrotic therapy may serve as a necessary addition to gene and cell therapies to treat DMD in the future. Therefore, understanding cellular and molecular mechanisms underlying muscle fibrogenesis associated with dystrophin deficiency is key to the development of effective antifibrotic therapies for DMD.

Original languageEnglish (US)
Pages (from-to)771-776
Number of pages6
JournalJournal of Neuropathology and Experimental Neurology
Volume69
Issue number8
DOIs
StatePublished - Aug 1 2010

Fingerprint

Duchenne Muscular Dystrophy
Fibrosis
Muscles
Dystrophin
Cell- and Tissue-Based Therapy
Genetic Therapy
Therapeutics
Inborn Genetic Diseases
Premature Mortality
Live Birth
Respiratory Insufficiency
Genes
Cicatrix
Regeneration
Stem Cells
Extremities
Heart Failure
Phenotype
Biopsy

Keywords

  • Antifibrotic therapy
  • Duchenne muscular dystrophy
  • Muscle fibrosis.

ASJC Scopus subject areas

  • Pathology and Forensic Medicine
  • Clinical Neurology
  • Neurology
  • Cellular and Molecular Neuroscience

Cite this

Targeting fibrosis in duchenne muscular dystrophy. / Zhou, Lan; Lu, Haiyan.

In: Journal of Neuropathology and Experimental Neurology, Vol. 69, No. 8, 01.08.2010, p. 771-776.

Research output: Contribution to journalReview article

Zhou, Lan ; Lu, Haiyan. / Targeting fibrosis in duchenne muscular dystrophy. In: Journal of Neuropathology and Experimental Neurology. 2010 ; Vol. 69, No. 8. pp. 771-776.
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