Clinico-pathological rescue of a model mouse of Huntington's disease by siRNA.
作者： Yu-Lai Wang , Wanzhao Liu , Etsuko Wada , Miho Murata , Keiji Wada
DOI: 10.1016/J.NEURES.2005.06.021
关键词:
摘要: Huntington's disease (HD) is an autosomal dominant inheritable neurodegenerative disorder currently without effective treatment. It caused by expanded polyglutamine (poly Q) tract in the corresponding protein, huntingtin (htt), and therefore suppressing expression brain neurons expected to delay onset mitigate severity of disease. Here, we have used small interfering RNAs (siRNAs) directed against gene repress transgenic mutant HD mouse model, R6/2. Results showed that intraventricular injection siRNAs at early postnatal period inhibited induced a decrease numbers sizes intranuclear inclusions striatal neurons. Treatments using this siRNA significantly prolonged model mice longevity, improved motor function slowed down loss body weight. This work suggests siRNA-based therapy promising as future treatment for HD.
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