Mice lacking the myotonic dystrophy protein kinase develop a late onset progressive myopathy

Nat Genet. 1996 Jul;13(3):325-35. doi: 10.1038/ng0796-325.

Abstract

Myotonic dystrophy (DM) is an autosomal dominant disorder resulting from the expansion of a CTG repeat in the 3' untranslated region of a putative protein kinase (DMPK). To elucidate the role of DMPK in DM pathogenesis we have developed Dmpk deficient (Dmpk-/-) mice. Dmpk-/-mice develop a late-onset, progressive skeletal myopathy that shares some pathological features with DM. Muscles from mature mice show variation in fibre size, increased fibre degeneration and fibrosis. Adult Dmpk-/-mice show ultrastructural changes in muscle and a 50% decrease in force generation compared to young mice. Our results indicate that DMPK may be necessary for the maintenance of skeletal muscle structure and function and suggest that a decrease in DMPK levels may contribute to DM pathology.

Publication types

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

MeSH terms

  • Animals
  • Electromyography
  • Female
  • Homozygote
  • Humans
  • Mice
  • Mice, Inbred C57BL
  • Mice, Transgenic
  • Muscle Fatigue
  • Muscle Fibers, Skeletal / chemistry
  • Muscle Fibers, Skeletal / pathology
  • Muscle, Skeletal / pathology*
  • Muscle, Skeletal / physiopathology
  • Muscle, Skeletal / ultrastructure
  • Mutation
  • Myotonic Dystrophy / genetics
  • Myotonic Dystrophy / pathology
  • Myotonin-Protein Kinase
  • Protein Serine-Threonine Kinases / biosynthesis
  • Protein Serine-Threonine Kinases / deficiency*
  • Protein Serine-Threonine Kinases / genetics
  • Regeneration

Substances

  • DMPK protein, human
  • DMPK protein, mouse
  • Myotonin-Protein Kinase
  • Protein Serine-Threonine Kinases