Wang Xin, Huang Timothy, Bu Guojun, Xu Huaxi
Fujian Provincial Key Laboratory of Neurodegenerative Disease and Aging Research, Institute of Neuroscience, College of Medicine, Xiamen University, Xiamen, Fujian 361102, China.
Mol Neurodegener. 2014 Aug 25;9:31. doi: 10.1186/1750-1326-9-31.
Intracellular protein trafficking plays an important role in neuronal function and survival. Protein misfolding is a common theme found in many neurodegenerative diseases, and intracellular trafficking machinery contributes to the pathological accumulation and clearance of misfolded proteins. Although neurodegenerative diseases exhibit distinct pathological features, abnormal endocytic trafficking is apparent in several neurodegenerative diseases, such as Alzheimer's disease (AD), Down syndrome (DS) and Parkinson's disease (PD). In this review, we will focus on protein sorting defects in three major neurodegenerative diseases, including AD, DS and PD. An important pathological feature of AD is the presence of extracellular senile plaques in the brain. Senile plaques are composed of β-amyloid (Aβ) peptide aggregates. Multiple lines of evidence demonstrate that over-production/aggregation of Aβ in the brain is a primary cause of AD and attenuation of Aβ generation has become a topic of extreme interest in AD research. Aβ is generated from β-amyloid precursor protein (APP) through sequential cleavage by β-secretase and the γ-secretase complex. Alternatively, APP can be cleaved by α-secretase within the Aβ domain to release soluble APPα which precludes Aβ generation. DS patients display a strikingly similar pathology to AD patients, including the generation of neuronal amyloid plaques. Moreover, all DS patients develop an AD-like neuropathology by their 40 s. Therefore, understanding the metabolism/processing of APP and how these underlying mechanisms may be pathologically compromised is crucial for future AD and DS therapeutic strategies. Evidence accumulated thus far reveals that synaptic vesicle regulation, endocytic trafficking, and lysosome-mediated autophagy are involved in increased susceptibility to PD. Here we review current knowledge of endosomal trafficking regulation in AD, DS and PD.
细胞内蛋白质运输在神经元功能和存活中起着重要作用。蛋白质错误折叠是许多神经退行性疾病中常见的现象,细胞内运输机制参与了错误折叠蛋白质的病理积累和清除。尽管神经退行性疾病表现出不同的病理特征,但异常的内吞运输在几种神经退行性疾病中很明显,如阿尔茨海默病(AD)、唐氏综合征(DS)和帕金森病(PD)。在这篇综述中,我们将重点关注三种主要神经退行性疾病中的蛋白质分选缺陷,包括AD、DS和PD。AD的一个重要病理特征是大脑中存在细胞外老年斑。老年斑由β-淀粉样蛋白(Aβ)肽聚集体组成。多条证据表明,大脑中Aβ的过度产生/聚集是AD的主要原因,减少Aβ的产生已成为AD研究中极其关注的一个话题。Aβ是由β-淀粉样前体蛋白(APP)通过β-分泌酶和γ-分泌酶复合物的顺序切割产生的。或者,APP可以在Aβ结构域内被α-分泌酶切割,释放可溶性APPα,从而阻止Aβ的产生。DS患者表现出与AD患者惊人相似的病理特征,包括神经元淀粉样斑块的形成。此外,所有DS患者在40多岁时都会出现类似AD的神经病理学特征。因此,了解APP的代谢/加工过程以及这些潜在机制如何在病理上受到损害,对于未来AD和DS的治疗策略至关重要。迄今为止积累的证据表明,突触小泡调节、内吞运输和溶酶体介导的自噬与PD易感性增加有关。在这里,我们综述了目前关于AD、DS和PD中内体运输调节的知识。