Overexpression of facioscapulohumeral muscular dystrophy region gene 1 causes primary defects in myogenic stem cells

J Cell Sci. 2013 May 15;126(Pt 10):2236-45. doi: 10.1242/jcs.121533. Epub 2013 Mar 22.

Abstract

Overexpression of facioscapulohumeral muscular dystrophy region gene 1 (FRG1) in mice, frogs and worms leads to muscular and vascular abnormalities. Nevertheless, the mechanism that follows FRG1 overexpression and finally leads to muscular defects is currently unknown. Here, we show that the earliest phenotype displayed by mice overexpressing FRG1 is a postnatal muscle-growth defect. Long before the development of muscular dystrophy, FRG1 mice also exhibit a muscle regeneration impairment. Ex vivo and in vivo experiments revealed that FRG1 overexpression causes myogenic stem cell activation and proliferative, clonogenic and differentiation defects. A comparative gene expression profiling of muscles from young pre-dystrophic wild-type and FRG1 mice identified differentially expressed genes in several gene categories and networks that could explain the emerging tissue and myogenic stem cell defects. Overall, our study provides new insights into the pathways regulated by FRG1 and suggests that muscle stem cell defects could contribute to the pathology of FRG1 mice.

Keywords: Epigenetics; Muscle stem cells; Muscular dystrophy.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Actin Cytoskeleton / metabolism
  • Adaptor Proteins, Signal Transducing / genetics
  • Adaptor Proteins, Signal Transducing / metabolism*
  • Animals
  • Autoimmune Diseases / metabolism*
  • Carrier Proteins / genetics
  • Carrier Proteins / metabolism*
  • Cell Line
  • Cell Surface Extensions / physiology*
  • Cytoskeletal Proteins / genetics
  • Cytoskeletal Proteins / metabolism*
  • Fatty Acid-Binding Proteins
  • Macrophages / physiology*
  • Mice
  • Multipotent Stem Cells / physiology*
  • Muscle Development / genetics
  • Muscular Dystrophy, Facioscapulohumeral / metabolism*
  • Protein Multimerization / genetics
  • Protein Structure, Tertiary / genetics
  • RNA, Small Interfering / genetics
  • Transgenes / genetics
  • Wiskott-Aldrich Syndrome Protein / metabolism

Substances

  • Adaptor Proteins, Signal Transducing
  • Carrier Proteins
  • Cytoskeletal Proteins
  • Fatty Acid-Binding Proteins
  • Fnbp1 protein, mouse
  • Pstpip2 protein, mouse
  • RNA, Small Interfering
  • Wiskott-Aldrich Syndrome Protein