Department of Pathology and Cell Biology, Columbia University, New York, NY 10032, United States.
Department of Pathology and Cell Biology, Columbia University, New York, NY 10032, United States; Department of Neurology, Columbia University, New York, NY 10032, United States.
Neurobiol Dis. 2019 Feb;122:16-22. doi: 10.1016/j.nbd.2018.08.010. Epub 2018 Aug 24.
The lysosome-mediated degradation pathway known as macroautophagy is the most versatile means through which cells can eliminate and recycle unwanted materials. Through both selective and non-selective means, macroautophagy can degrade a wide range of cargoes from bulk cytosol to organelles and aggregated proteins. Although studies of disorders such as Parkinson's disease and Amyotrophic Lateral Sclerosis suggest that autophagic and lysosomal dysfunction directly contributes to disease, this had not been the case for the polyglutamine disorder Huntington's disease (HD), for which there was little indication of a disruption in the autophagic-lysosomal system. This supported the possibility of targeting autophagy as a much needed therapeutic approach to combat this disease. Possibly challenging this view, however, are a recent set of studies suggesting that the protein affected in Huntington's disease, huntingtin, might mechanistically contribute to macroautophagy. In this review, we will explore how autophagy might impact or be impacted by HD pathogenesis, and whether a therapeutic approach centering on autophagy may be possible for this yet incurable disease.
溶酶体介导的降解途径,即所谓的巨自噬,是细胞消除和回收不需要的物质的最通用的方法。通过选择性和非选择性的方式,巨自噬可以降解从细胞质到细胞器和聚集体蛋白等各种物质。虽然对帕金森病和肌萎缩侧索硬化症等疾病的研究表明,自噬和溶酶体功能障碍直接导致疾病,但亨廷顿病(HD)这种多聚谷氨酰胺疾病并非如此,因为自噬溶酶体系统几乎没有受到干扰的迹象。这支持了将自噬作为治疗这种疾病的一种急需的治疗方法的可能性。然而,最近的一组研究可能挑战了这一观点,这些研究表明,亨廷顿病中受影响的蛋白质,亨廷顿蛋白,可能在机制上有助于巨自噬。在这篇综述中,我们将探讨自噬可能如何影响或受 HD 发病机制的影响,以及以自噬为中心的治疗方法是否可能对这种仍然无法治愈的疾病有效。