Trinucleotide repeats in neurogenetic disorders

Annu Rev Neurosci. 1996:19:79-107. doi: 10.1146/annurev.ne.19.030196.000455.

Abstract

Trinucleotide repeat expansion is increasingly recognized as a cause of neurogenetic diseases. To date, seven diseases have been identified as expanded repeat disorders: the fragile X syndrome of mental retardation both FRAXA and FRAXE loci), myotonic dystrophy, X-linked spinal and bulbar muscular atrophy, Huntington's disease, spinocerebellar ataxia type I, dentatorubral-pallidoluysian atrophy, and Machado-Joseph disease. All are neurologic disorders, affecting one or more regions of the neuraxis. Moreover, five of the seven (the last five above) are progressive neurodegenerative disorders whose strikingly similar mutations suggest a common mechanism of neuronal degeneration. In this article we discuss specific characteristics of each trinucleotide repeat disease, review their shared clinical and genetic features, and address possible molecular mechanisms underlying the neuropathology in each disease. Particular attention is paid to the neurodegenerative diseases, all of which are caused by CAG repeats encoding polyglutamine tracts in the disease gene protein.

Publication types

  • Research Support, Non-U.S. Gov't
  • Research Support, U.S. Gov't, P.H.S.
  • Review

MeSH terms

  • Central Nervous System Diseases / genetics*
  • Fragile X Syndrome / genetics
  • Humans
  • Huntington Disease / genetics
  • Intellectual Disability / genetics
  • Machado-Joseph Disease / genetics
  • Muscular Atrophy / genetics
  • Myotonic Dystrophy / genetics
  • Spinocerebellar Degenerations / genetics
  • Trinucleotide Repeats*