Huntington's disease (HD) is a late-onset neurodegenerative disorder caused by a polyglutamine expansion in huntingtin, a protein of unknown function. Transgenic models expressing a portion or full-length human huntingtin have been generated to unravel the mechanism through which the mutation causes the symptoms and the selective cell death characteristic of HD. We review here advances in the understanding of HD made possible by transgenic models and the means by which they implicate polyglutamine aggregation in the pathology of triplet repeat disorders.
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