Convergent pathogenic pathways in Alzheimer's and Huntington's diseases: shared targets for drug development.

Convergent pathogenic pathways in Alzheimer's and Huntington's diseases: shared targets for drug development.
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DOI:
10.1038/nrd3556
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发表时间:
2011-10-21
影响因子:
120.1
通讯作者:
Hayden, Michael R.
Hayden, Michael R.
中科院分区:
医学1区
文献类型:
--
作者:
Ehrnhoefer, Dagmar E.;Wong, Bibiana K. Y.;Hayden, Michael R.

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以阿尔茨海默病和亨廷顿病为代表的神经退行性疾病的特点是进行性的神经精神功能障碍和特定神经元亚型的丢失。尽管在确切的病理部位和临床特征上存在差异,但在发病机制和致病途径上可以观察到相当大的相似之处。这些共同的机制增加了药物开发的共同治疗靶点的可能性。亨廷顿病具有单基因病因和准确识别显性前突变携带者的可能性,可被用作阿尔茨海默病的“模型”,以测试针对共同致病途径的治疗干预的有效性。
Neurodegenerative diseases exemplified by Alzheimer’s and Huntington disease are characterized by the progressive neuropsychiatric dysfunction and loss of specific neuronal subtypes. Even though there are differences in the exact sites of pathology and clinical profiles only partially overlap, considerable similarities in disease mechanisms and pathogenic pathways can be observed. These shared mechanisms raise the possibility of common therapeutic targets for drug development. Huntington disease with a monogenic cause and the possibility to accurately identify pre-manifest mutation carriers could be exploited as a ‘model’ for Alzheimer’s disease to test the efficacy of therapeutic interventions targeting shared pathogenic pathways.
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