Abstract

Despite important advances in understanding and elucidating the molecular and mechanistic pathways that mediate progression in Huntington's disease (HD), effective pharmacotherapy remains elusive. Insights into disease pathogenesis have come from studies using tissue culture, yeast, Caenorhabditis elegans, Drosophila melanogaster and transgenic mouse models. Here, we present a brief overview of HD pathogenesis and discuss the efficacy of therapeutic agents in transgenic mouse models of HD. We conclude by considering issues that affect the translation of findings in transgenic mouse models of HD to human clinical trials.

Author affiliations

1. Department of Neurology and Neuroscience, Weill Medical College of Cornell University, Room F610, 525 East 68th Street, New York, NY 10021 USA.
2. Geriatric Research Education and Clinical Center, Bedford VA Medical Center, Bedford, Massachusetts and the Departments of Neurology, Pathology and Psychiatry, Boston University School of Medicine, Boston, Massachusetts 02118, USA.

Correspondence to: M. Flint Beal¹ Email: fbeal@med.cornell.edu

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