Murphy Robert C, Messer Anne
Wadsworth Center/David Axelrod Institute, New York State Department of Health and Department of Biomedical Sciences, University at Albany, P.O. Box 22002, Albany, NY 12201-2002, USA.
Brain Res Mol Brain Res. 2004 Feb 5;121(1-2):141-5. doi: 10.1016/j.molbrainres.2003.11.011.
Huntington's disease (HD) is a progressive, hereditary, neurodegenerative disorder caused by an expanded polyglutamine tract in huntingtin protein, leading to misfolding and abnormal protein-protein interactions. Reducing the initial misfolding should lead to decreased pathogenesis. We show that malonate stress increases the number of dead or dying cells when organotypic slice cultures are transduced to express pathological-length huntingtin fragments. Co-transfected anti-HD single-chain Fv (sFv) intrabodies can reverse this HD-specific increase in malonate-induced morbidity.
亨廷顿舞蹈症（HD）是一种进行性、遗传性神经退行性疾病，由亨廷顿蛋白中多聚谷氨酰胺序列的扩展引起，导致蛋白质错误折叠和异常的蛋白质-蛋白质相互作用。减少初始错误折叠应能降低发病机制。我们发现，当器官型脑片培养物被转导以表达病理长度的亨廷顿片段时，丙二酸应激会增加死亡或濒死细胞的数量。共转染的抗HD单链Fv（sFv）胞内抗体可以逆转丙二酸诱导的HD特异性发病率增加。
