CAG repeat expansion in the TATA box-binding protein gene causes autosomal dominant cerebellar ataxia

Brain. 2001 Oct;124(Pt 10):1939-47. doi: 10.1093/brain/124.10.1939.

Abstract

At least 13 loci responsible for autosomal dominant cerebellar ataxia (ADCA) have been identified. Spinocerebellar ataxia 1, 2, 3, 6 and 7 are caused by translated CAG repeat expansions. However, in France, >30% of ADCAs are not explained by the known genes. Recently, analysis of the TATA box-binding protein (TBP) gene, one of the transcription factors known to contain a CAG/CAA repeat, in patients with progressive cerebellar ataxia revealed one sporadic case with 63 repeats. We examined this gene in 162 index cases with ADCA. An expanded repeat with 46 repeat units was detected in a single index case from Belgium. In this family, two affected members and six unaffected, but at-risk, individuals carried expanded alleles. Interestingly, the expanded repeat was stable during transmission. The main clinical features in six patients were cerebellar ataxia, dementia and behavioural disturbances with onset in their fourth to sixth decade. The main neuropathological finding was severe neuronal loss and gliosis in the Purkinje cell layer. Immunohistochemical analysis showed neuronal intranuclear inclusions containing expanded polyglutamine, indicating that this disease shares several features with other polyglutamine diseases. This study demonstrates that CAG/CAA repeat expansion in the TBP gene causes ADCA with dementia and/or psychiatric manifestations.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adult
  • Aged
  • Atrophy
  • Brain / pathology
  • Cerebellar Ataxia / genetics*
  • Cerebellar Ataxia / pathology
  • DNA-Binding Proteins / genetics*
  • Female
  • Gliosis / genetics
  • Gliosis / pathology
  • Humans
  • Immunohistochemistry
  • Male
  • Middle Aged
  • Pedigree
  • TATA Box / genetics*
  • TATA-Box Binding Protein
  • Transcription Factors / genetics*
  • Trinucleotide Repeat Expansion / genetics*

Substances

  • DNA-Binding Proteins
  • TATA-Box Binding Protein
  • Transcription Factors