East Hospital, Tongji University School of Medicine, Shanghai, China.
Department of Neuroscience, Sheffield Institute for Translational Neuroscience (SITraN), University of Sheffield, Sheffield, UK.
Cell Death Dis. 2019 Feb 4;10(2):105. doi: 10.1038/s41419-019-1313-y.
Parkinson's disease (PD) is an aging-related degenerative disorder arisen from the loss of dopaminergic neurons in substantia nigra. Although many genetic mutations have been implicated to be genetically linked to PD, the low incidence of familial PD carried with mutations suggests that there must be other factors such as oxidative stress, mitochondrial dysfunction, accumulation of misfolded proteins, and enhanced inflammation, which are contributable to the pathophysiology of PD. The major efforts of current research have been devoted to unravel the toxic effect of multiple factors, which directly cause the degeneration of dopaminergic neurons in adulthood. Until recently, several studies have demonstrated that NSCs had compromised proliferation and differentiation capacity in PD animal models or PD patient-derived iPS models, suggesting that the pathology of PD may be rooted in some cellular aberrations at early developmental stage but the mechanism remains to be elusive. Based on the early-onset PD patient-specific iPSCs, we found that PD-patient iPSC-derived NSCs were more susceptible to stress and became functionally compromised by radiation or oxidative insults. We further unraveled that stress-induced SIRT1 downregulation leading to autophagic dysfunction, which were responsible for these deficits in PD-NSCs. Mechanistically, we demonstrated that stress-induced activation of p38 MAPK suppressed SIRT1 expression, which in turn augmented the acetylation of multiple ATG proteins of autophagic complex and eventually led to autophagic deficits. Our studies suggest that early developmental deficits may, at least partially, contribute to the pathology of PD and provide a new avenue for developing better therapeutic interventions to PD.
帕金森病(PD)是一种与衰老相关的退行性疾病,源于黑质中多巴胺能神经元的丧失。虽然许多基因突变已被认为与 PD 有遗传关联,但携带突变的家族性 PD 的发病率较低表明,一定还有其他因素,如氧化应激、线粒体功能障碍、错误折叠蛋白的积累和炎症增强,这些因素与 PD 的病理生理学有关。目前的研究主要致力于揭示多种因素的毒性作用,这些因素直接导致成年多巴胺能神经元的退化。直到最近,几项研究表明,PD 动物模型或 PD 患者来源的 iPS 模型中的 NSCs 增殖和分化能力受损,这表明 PD 的病理学可能源于早期发育阶段的某些细胞异常,但机制仍不清楚。基于早发性 PD 患者特异性 iPSCs,我们发现 PD 患者来源的 iPSC 衍生的 NSCs 对压力更敏感,并因辐射或氧化损伤而功能受损。我们进一步揭示了应激诱导的 SIRT1 下调导致自噬功能障碍,这是 PD-NSCs 这些缺陷的原因。在机制上,我们证明应激诱导的 p38 MAPK 激活抑制了 SIRT1 的表达,进而增加了自噬体复合物中多个 ATG 蛋白的乙酰化,最终导致自噬缺陷。我们的研究表明,早期发育缺陷可能至少部分导致 PD 的病理学,并为开发更好的 PD 治疗干预措施提供了新途径。