Sari Youssef
University of Toledo, College of Pharmacy & Pharmaceutical Sciences, Department of Pharmacology, OH 43614, USA.
Recent Pat CNS Drug Discov. 2011 May 1;6(2):80-90. doi: 10.2174/157488911795933884.
Huntington's disease (HD) is an autosomal dominant inherited and progressive neurodegenerative disorder with motor dysfunction and cognitive deficits. Although there are no treatments to delay the appearance and the progression of HD, there are potential drugs currently in preclinical and clinical trials that are focused on HD therapy. The signaling pathways involved in HD are not yet clearly elucidated; however, expression of mutant huntingtin protein is considered a key factor in the induction and/or progression of HD. The demonstration that the onset and progression of HD in models of transgenic mice, in particular, are delayed or improved by the application of neurotrophic factors has emphasized their importance in neuroprotection in HD. In addition, other compounds targeting the HD gene or mutant huntingtin protein are currently in preclinical and clinical testing and may show promising neuroprotective effects. There are current patented drugs that are currently being considered as potential therapeutics for HD. These patented drugs may provide promising therapy for HD.
亨廷顿舞蹈症（HD）是一种常染色体显性遗传的渐进性神经退行性疾病，伴有运动功能障碍和认知缺陷。尽管尚无延缓HD出现和进展的治疗方法，但目前有一些处于临床前和临床试验阶段的潜在药物专注于HD治疗。HD所涉及的信号通路尚未完全阐明；然而，突变型亨廷顿蛋白的表达被认为是HD诱导和/或进展的关键因素。特别是在转基因小鼠模型中，应用神经营养因子可延缓或改善HD的发病和进展，这一结果凸显了它们在HD神经保护中的重要性。此外，目前还有其他针对HD基因或突变型亨廷顿蛋白的化合物正处于临床前和临床试验阶段，可能会显示出有前景的神经保护作用。目前有一些专利药物正被考虑作为HD的潜在治疗方法。这些专利药物可能为HD提供有前景的治疗方案。
