Identification of a novel mutation in a Korean patient with oculopharyngeal muscular dystrophy

J Clin Neurosci. 2007 Jan;14(1):89-92. doi: 10.1016/j.jocn.2005.12.036.

Abstract

Oculopharyngeal muscular dystrophy (OPMD) is a late-onset muscle disorder characterized by progressive dysphagia and bilateral ptosis. Mutations in the polyadenylate binding protein nuclear 1 (PABPN1) gene have been found to cause OPMD. The typical mutation is a stable trinucleotide repeat expansion in the first exon of the PABPN1 gene, in which (GCG)(6) is the normal repeat length. We investigated a Korean patient with OPMD and identified a novel mutation: a heterozygous insertion of a 9-bp sequence [(GCG)(GCA)(GCA); c.27_28insGCGGCAGCA] instead of the (GCG) repeat expansion, resulting in an in-frame insertion of three alanines (p.A10insAAA). To the best of our knowledge, this is the first report of a genetically confirmed case of OPMD in Korea.

Publication types

  • Case Reports

MeSH terms

  • Blepharoptosis / etiology
  • DNA Transposable Elements
  • Deglutition Disorders / etiology
  • Deglutition Disorders / therapy
  • Exons / genetics
  • Humans
  • Korea
  • Male
  • Middle Aged
  • Muscular Dystrophy, Oculopharyngeal / complications
  • Muscular Dystrophy, Oculopharyngeal / diagnosis
  • Muscular Dystrophy, Oculopharyngeal / genetics*
  • Mutation / physiology*
  • Pedigree
  • Poly(A)-Binding Protein II / genetics*
  • Trinucleotide Repeats

Substances

  • DNA Transposable Elements
  • Poly(A)-Binding Protein II