[The clinical-genealogic and molecular-genetic characteristics of oculopharyngeal muscular dystrophy in the Republic of Sakha (Yakutia)]

Zh Nevrol Psikhiatr Im S S Korsakova. 2008;108(6):52-60.
[Article in Russian]

Abstract

The clinical-genealogic and molecular-genetic investigation of oculopharyngeal muscular dystrophy (OPMD) in the Republic of Sakha (Yakutia) was performed. It was investigated 33 unrelated Yakut families with 38 patients and 2 russian families with 2 patients and 59 their healthy relatives as well. The high clinical polymorphism of disease was found in patients with OPMD. The mutation in exon 1 of the PABPN1 gene resulting in the expansion of GCG-repeats up to 10 is revealed. Using direct sequencing of the PABPN1 gene in 17 families (16 Yakut, 1 Russian), we identified a type of this mutation as an insertion of 4 GCG-repeats. Frequency of OPMD in the Yakut population is 1:11 680 that is 10-20 times higher comparing to european populations. This is a first report on the patients with OPMD from the Republic of Sakha with diagnosis confirmed by molecular-genetic analysis.

MeSH terms

  • Adult
  • Aged
  • Catchment Area, Health
  • Exons / genetics
  • Female
  • Humans
  • Male
  • Middle Aged
  • Muscular Dystrophy, Oculopharyngeal / epidemiology*
  • Muscular Dystrophy, Oculopharyngeal / genetics*
  • Pedigree
  • Point Mutation / genetics
  • Poly(A)-Binding Protein II / genetics*
  • Polymorphism, Genetic / genetics
  • Russia / epidemiology
  • Trinucleotide Repeat Expansion / genetics

Substances

  • Poly(A)-Binding Protein II