Centre for Molecular Medicine and Therapeutics, Department of Medical Genetics, Child & Family Research Institute, University of British Columbia, 950 West 28th Avenue, Vancouver, British Columbia V5Z 4H4, Canada.
Nat Rev Drug Discov. 2011 Oct 21;10(11):853-67. doi: 10.1038/nrd3556.
Neurodegenerative diseases, exemplified by Alzheimer's disease and Huntington's disease, are characterized by progressive neuropsychiatric dysfunction and loss of specific neuronal subtypes. Although there are differences in the exact sites of pathology, and the clinical profiles of these two conditions only partially overlap, considerable similarities in disease mechanisms and pathogenic pathways can be observed. These shared mechanisms raise the possibility of exploiting common therapeutic targets for drug development. As Huntington's disease has a monogenic cause, it is possible to accurately identify individuals who carry the Huntington's disease mutation but do not yet manifest symptoms. These individuals could act as a model for Alzheimer's disease to test therapeutic interventions that target shared pathogenic pathways.
神经退行性疾病,以阿尔茨海默病和亨廷顿病为代表,其特征是进行性神经精神功能障碍和特定神经元亚型的丧失。尽管在病理学的确切部位存在差异,并且这两种疾病的临床特征仅部分重叠,但在疾病机制和发病途径方面存在相当多的相似之处。这些共同的机制提出了利用共同的治疗靶点开发药物的可能性。由于亨廷顿病的病因是单基因的,因此可以准确识别携带亨廷顿病突变但尚未出现症状的个体。这些个体可以作为阿尔茨海默病的模型,以测试针对共同发病途径的治疗干预措施。