Martini-Stoica Heidi, Xu Yin, Ballabio Andrea, Zheng Hui
Huffington Center on Aging, Baylor College of Medicine, Houston, TX, USA; Interdepartmental Program in Translational Biology and Molecular Medicine, Baylor College of Medicine, Houston, TX, USA; Medical Scientist Training Program, Baylor College of Medicine, Houston, TX, USA.
Huffington Center on Aging, Baylor College of Medicine, Houston, TX, USA.
Trends Neurosci. 2016 Apr;39(4):221-234. doi: 10.1016/j.tins.2016.02.002. Epub 2016 Mar 9.
The autophagy-lysosomal pathway (ALP) is involved in the degradation of long-lived proteins. Deficits in the ALP result in protein aggregation, the generation of toxic protein species, and accumulation of dysfunctional organelles, which are hallmarks of Alzheimer's disease (AD), Parkinson's disease (PD), Huntington's disease (HD), and prion disease. Decades of research have therefore focused on enhancing the ALP in neurodegenerative diseases. More recently, transcription factor EB (TFEB), a major regulator of autophagy and lysosomal biogenesis, has emerged as a leading factor in addressing disease pathology. We review the regulation of the ALP and TFEB and their impact on neurodegenerative diseases. We also offer our perspective on the complex role of autophagy and TFEB in disease pathogenesis and its therapeutic implications through the examination of prion disease.
自噬-溶酶体途径(ALP)参与长寿蛋白的降解。ALP功能缺陷会导致蛋白质聚集、有毒蛋白质种类的产生以及功能失调细胞器的积累,这些都是阿尔茨海默病(AD)、帕金森病(PD)、亨廷顿舞蹈病(HD)和朊病毒病的特征。因此,数十年来的研究都集中在增强神经退行性疾病中的ALP。最近,转录因子EB(TFEB)作为自噬和溶酶体生物发生的主要调节因子,已成为解决疾病病理问题的关键因素。我们综述了ALP和TFEB的调节及其对神经退行性疾病的影响。我们还通过对朊病毒病的研究,就自噬和TFEB在疾病发病机制中的复杂作用及其治疗意义提出了我们的观点。