Unstable expansion of CAG repeat in hereditary dentatorubral-pallidoluysian atrophy (DRPLA)

Nat Genet. 1994 Jan;6(1):9-13. doi: 10.1038/ng0194-9.

Abstract

Hereditary dentatorubral-pallidoluysian atrophy (DRPLA) is an autosomal dominant neurologic disorder characterized by variable combinations of myoclonus, epilepsy, cerebellar ataxia, choreoathetosis and dementia. By specifically searching published brain cDNA sequences for the presence of CAG repeats we identified unstable expansion of a CAG in a gene on chromosome 12 in all the 22 DRPLA patients examined. A good correlation between the size of the CAG repeat expansion and the ages of disease onset is found in this group. Patients with earlier onset tended to have a phenotype of progressive myoclonus epilepsy and larger expansions. We propose that the wide variety of clinical manifestations of DRPLA can now be explained by the variable unstable expansion of the CAG repeat.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Adult
  • Amino Acid Sequence
  • Base Sequence
  • Brain / pathology
  • Cerebellar Ataxia / genetics
  • Child
  • DNA, Complementary / genetics
  • Dementia / genetics
  • Epilepsies, Myoclonic / genetics
  • Female
  • Humans
  • Male
  • Middle Aged
  • Molecular Sequence Data
  • Nervous System Diseases / genetics*
  • Nervous System Diseases / pathology
  • Oligodeoxyribonucleotides / genetics
  • Pedigree
  • Repetitive Sequences, Nucleic Acid*

Substances

  • DNA, Complementary
  • Oligodeoxyribonucleotides