Xiao Han, Tang Jianguang, Hu Zhiping, Tan Jieqiong, Tang Beisha, Jiang Zheng
Department of Neurology, the Second Xiangya Hospital, Central South University, Changsha, Hunan, 410011 P.R.China.
Zhonghua Yi Xue Yi Chuan Xue Za Zhi. 2010 Feb;27(1):23-8. doi: 10.3760/cma.j.issn.1003-9406.2010.01.005.
To investigate the role of autophagy on the pathogenesis of spinocerebellar ataxia 3/Machado-Joseph disease (SCA3/MJD).
HEK293 cells expressing polyglutamine-expanded ataxin-3 were used as cell model for SCA3/MJD. The level of polyglutamine-expanded ataxin-3 was detected after cells were treated with different inhibitors or inducer of autophagy.
Inhibition of autophagy increased aggregate formation and cell death in HEK293 cells expressing mutated ataxin-3, and vice versa.
The data suggested that autophagy is involved in the degradation of mutant ataxin-3, resulting in a decrease in the proportions of aggregate-containing cells and cell death in HEK293 cells expressing polyglutamine-expanded ataxin-3. It is possible that autophagy may be applied as a potential therapeutic approach for SCA3/MJD.
研究自噬在脊髓小脑共济失调3型/马查多-约瑟夫病（SCA3/MJD）发病机制中的作用。
将表达多聚谷氨酰胺扩展型ataxin-3的HEK293细胞用作SCA3/MJD的细胞模型。在用不同的自噬抑制剂或诱导剂处理细胞后，检测多聚谷氨酰胺扩展型ataxin-3的水平。
自噬抑制增加了表达突变型ataxin-3的HEK293细胞中的聚集体形成和细胞死亡，反之亦然。
数据表明自噬参与突变型ataxin-3的降解，导致表达多聚谷氨酰胺扩展型ataxin-3的HEK293细胞中含聚集体细胞的比例和细胞死亡减少。自噬有可能作为SCA3/MJD的一种潜在治疗方法。
