作者: M. Flint Beal , Robert J. Ferrante
DOI: 10.1038/NRN1386
关键词:
摘要: Despite important advances in understanding and elucidating the molecular mechanistic pathways that mediate progression Huntington's disease (HD), effective pharmacotherapy remains elusive. Insights into pathogenesis have come from studies using tissue culture, yeast, Caenorhabditis elegans, Drosophila melanogaster transgenic mouse models. Here, we present a brief overview of HD discuss efficacy therapeutic agents models HD. We conclude by considering issues affect translation findings to human clinical trials.