Conway K A, Harper J D, Lansbury P T
Center for Neurologic Diseases, Brigham and Women's Hospital and Department of Neurology, Harvard Medical School, Boston, Massachusetts 02115, USA.
Nat Med. 1998 Nov;4(11):1318-20. doi: 10.1038/3311.
Two mutations in the gene encoding alpha-synuclein have been linked to early-onset Parkinson's disease (PD). alpha-Synuclein is a component of Lewy bodies, the fibrous cytoplasmic inclusions characteristic of nigral dopaminergic neurons in the PD brain. This connection between genetics and pathology suggests that the alpha-synuclein mutations may promote PD pathogenesis by accelerating Lewy body formation. To test this, we studied alpha-synuclein folding and aggregation in vitro, in the absence of other Lewy body-associated molecules. We demonstrate here that both mutant forms of alpha-synuclein (A53T and A30P) are, like wild-type alpha-synuclein (WT), disordered in dilute solution. However, at higher concentrations, Lewy body-like fibrils and discrete spherical assemblies are formed; most rapidly by A53T. Thus, mutation-induced acceleration of alpha-synuclein fibril formation may contribute to the early onset of familial PD.
编码α-突触核蛋白的基因中的两种突变已与早发性帕金森病(PD)相关联。α-突触核蛋白是路易小体的一个组成部分,路易小体是帕金森病大脑中黑质多巴胺能神经元特有的纤维状细胞质内含物。遗传学与病理学之间的这种联系表明,α-突触核蛋白突变可能通过加速路易小体形成来促进帕金森病的发病机制。为了验证这一点,我们在没有其他路易小体相关分子的情况下,在体外研究了α-突触核蛋白的折叠和聚集。我们在此证明,α-突触核蛋白的两种突变形式(A53T和A30P)与野生型α-突触核蛋白(WT)一样,在稀溶液中是无序的。然而,在较高浓度下,会形成路易小体样纤维和离散的球形聚集体;A53T形成得最快。因此,突变诱导的α-突触核蛋白纤维形成加速可能导致家族性帕金森病的早发。