Myoclonus-dystonia due to genomic deletions in the epsilon-sarcoglycan gene

Ann Neurol. 2005 Nov;58(5):792-7. doi: 10.1002/ana.20661.

Abstract

Direct genomic DNA sequencing fails to detect epsilon-sarcoglycan (SGCE) mutations in up to 30% of familial myoclonus-dystonia (M-D) cases. We identified novel large heterozygous deletions of SGCE exon 5 or exon 6 in two M-D pedigrees. Like nonsense mutations, exon rearrangements result in the generation of premature stop codons downstream of the deleted exon. SGCE exon dosage assays may identify additional families with SGCE mutation and thus reduce "genetic heterogeneity."

Publication types

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

MeSH terms

  • Adolescent
  • Adult
  • DNA Mutational Analysis
  • Dystonic Disorders / genetics*
  • Dystonic Disorders / physiopathology
  • Exons
  • Family Health
  • Female
  • Gene Deletion*
  • Gene Dosage
  • Humans
  • Male
  • Myoclonus / genetics*
  • Pedigree
  • RNA, Messenger / biosynthesis
  • Reverse Transcriptase Polymerase Chain Reaction
  • Sarcoglycans / genetics*

Substances

  • RNA, Messenger
  • Sarcoglycans