Missense mutations in desmin associated with familial cardiac and skeletal myopathy

Nat Genet. 1998 Aug;19(4):402-3. doi: 10.1038/1300.

Abstract

Desmin-related myopathy (OMIM 601419) is a familial disorder characterized by skeletal muscle weakness associated with cardiac conduction blocks, arrhythmias and restrictive heart failure, and by intracytoplasmic accumulation of desmin-reactive deposits in cardiac and skeletal muscle cells. The underlying molecular mechanisms are unknown. Involvement of the desmin gene (DES) has been excluded in three families diagnosed with desmin-related myopathy. We report two new families with desmin-related cardioskeletal myopathy associated with mutations in the highly conserved carboxy-terminal end of the desmin rod domain. A heterozygous A337P mutation was identified in a family with an adult-onset skeletal myopathy and mild cardiac involvement. Compound heterozygosity for two other mutations, A360P and N393I, was detected in a second family characterized by childhood-onset aggressive course of cardiac and skeletal myopathy.

MeSH terms

  • Adult
  • Age of Onset
  • Aged
  • Cardiomyopathies / genetics*
  • Child
  • Child, Preschool
  • DNA Mutational Analysis
  • Desmin / genetics*
  • Female
  • Heterozygote
  • Humans
  • Male
  • Middle Aged
  • Molecular Sequence Data
  • Muscular Diseases / genetics*
  • Pedigree
  • Point Mutation / genetics*

Substances

  • Desmin

Associated data

  • GENBANK/AF055081
  • GENBANK/AF055082
  • GENBANK/AF055083
  • GENBANK/U59167