Zhang Li, Jiang Hong
Department of Neurology, Xiangya Hospital, Central South University, Changsha, Hunan 410008, P.R. China.
Zhonghua Yi Xue Yi Chuan Xue Za Zhi. 2013 Dec;30(6):683-6. doi: 10.3760/cma.j.issn.1003-9406.2013.06.011.
Polyglutamine(PolyQ) diseases comprise a group of inherited neurodegenerative disorders with significant clinical and genetic heterogeneity. Although they share a common mechanism involving dynamic expansion of CAG trinucleotide repeats, their clinical features may vary and there has been no specific treatment. Recently, much attention had been attracted to microRNAs which, as a new type of posttranscription regulatory factor, have proven to significantly affect the progress of PolyQ disease. This review will focus on the roles of microRNAs in the pathogenesis of PolyQ diseases and their potential use for therapy.
聚谷氨酰胺(PolyQ)疾病是一组具有显著临床和遗传异质性的遗传性神经退行性疾病。尽管它们具有涉及CAG三核苷酸重复序列动态扩增的共同机制,但其临床特征可能有所不同,且尚无特异性治疗方法。最近,作为一种新型转录后调控因子的微小RNA(microRNA)已引起广泛关注,事实证明其能显著影响聚谷氨酰胺疾病的进展。本综述将聚焦于微小RNA在聚谷氨酰胺疾病发病机制中的作用及其潜在的治疗用途。