Cell based therapy for Duchenne muscular dystrophy

J Cell Physiol. 2009 Dec;221(3):526-34. doi: 10.1002/jcp.21895.

Abstract

Mutations in the dystrophin gene cause an X-linked genetic disorder: Duchenne muscular dystrophy (DMD). Stem cell therapy is an attractive method to treat DMD because a small number of cells are required to obtain a therapeutic effect. Here, we discussed about multiple types of myogenic stem cells and their possible use to treat DMD. The identification of a stem cell population providing efficient muscle regeneration is critical for the progression of cell therapy for DMD. We speculated that the most promising possibility for the treatment of DMD is a combination of different approaches, such as gene and stem cell therapy.

Publication types

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

MeSH terms

  • AC133 Antigen
  • Animals
  • Antigens, CD / analysis
  • Genetic Therapy / methods*
  • Glycoproteins / analysis
  • Humans
  • Muscular Dystrophy, Duchenne / genetics
  • Muscular Dystrophy, Duchenne / pathology
  • Muscular Dystrophy, Duchenne / therapy*
  • Peptides / analysis
  • Pericytes / cytology
  • Satellite Cells, Skeletal Muscle / cytology
  • Satellite Cells, Skeletal Muscle / transplantation
  • Stem Cell Transplantation*
  • Stem Cells / cytology
  • Stem Cells / metabolism

Substances

  • AC133 Antigen
  • Antigens, CD
  • Glycoproteins
  • Peptides