Abundant expression and cytoplasmic aggregations of [alpha]1A voltage-dependent calcium channel protein associated with neurodegeneration in spinocerebellar ataxia type 6

Hum Mol Genet. 1999 Jul;8(7):1185-93. doi: 10.1093/hmg/8.7.1185.

Abstract

Spinocerebellar ataxia type 6 (SCA6) is one of the eight neurodegenerative diseases caused by a tri-nucleotide (CAG) repeat expansion coding polyglutamine (CAG repeat/polyglutamine diseases) and is characterized by late onset autosomal dominant cerebellar ataxia and predominant loss of cerebellar Purkinje cells. Although the causative, small and stable CAG repeat expansion for this disease has been identified in the [alpha]1A voltage-dependent calcium channel gene (CACNA1A), the mechanism which leads to predominant Purkinje cell degeneration is totally unknown. In this study, we show that the calcium channel mRNA/protein containing the CAG repeat/polyglutamine tract is most intensely expressed in Purkinje cells of human brains. In SCA6 brains, numerous oval or rod-shaped aggregates were seen exclusively in the cytoplasm of Purkinje cells. These cytoplasmic inclusions were not ubiquitinated, which contrasts with the neuronal intra-nuclear inclusions of other CAG repeat/polyglutamine diseases. In cultured cells, formation of perinuclear aggregates of the channel protein and apoptotic cell death were seen when transfected with full-length CACNA1A coding an expanded polyglutamine tract. The present study indicates that the mechanism of neurodegeneration in SCA6 is associated with cytoplasmic aggregations of the [alpha]1A calcium channel protein caused by a small CAG repeat/polyglutamine expansion in CACNA1A.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Apoptosis
  • Brain / metabolism*
  • Brain / pathology
  • Calcium Channels / biosynthesis*
  • Calcium Channels / genetics
  • Cells, Cultured
  • Cytoplasm / metabolism
  • Humans
  • PC12 Cells
  • Peptides / metabolism
  • Purkinje Cells
  • RNA, Messenger / biosynthesis
  • Rats
  • Spinocerebellar Degenerations / genetics
  • Spinocerebellar Degenerations / metabolism*
  • Spinocerebellar Degenerations / pathology
  • Trinucleotide Repeat Expansion / genetics

Substances

  • CACNA1A protein, human
  • Cacna1a protein, rat
  • Calcium Channels
  • Peptides
  • RNA, Messenger
  • polyglutamine