Ibáñez Pablo, Lesage Suzanne, Lohmann Ebba, Thobois Stéphane, De Michele Giuseppe, Borg Michel, Agid Yves, Dürr Alexandra, Brice Alexis
INSERM U679, Neurologie et Thérapeutique Expérimentale, CHU Pitié-Salpêtrière, Paris, France.
Brain. 2006 Mar;129(Pt 3):686-94. doi: 10.1093/brain/awl005. Epub 2006 Jan 9.
Parkinson's disease is a frequent disorder caused primarily by the loss of dopaminergic neurons of the substantia nigra. Mutations in the PTEN-induced kinase (PINK1) gene, in addition to those in parkin and DJ-1, have been found in families with recessive early-onset Parkinson's disease. We screened for parkin and PINK1 mutations in a panel of 177 autosomal recessive Parkinson's disease families with ages at onset < or =60 years, mostly from Europe. In 7 unrelated families, we identified 10 pathogenic PINK1 mutations (5 missense, 2 nonsense and 3 frameshift deletion mutations), 8 of which were novel. All the mutations were in the homozygous or compound heterozygous states. Interestingly, pseudo-dominant inheritance was observed in a family with two different mutations. The clinical characteristics of 12 PINK1 patients and 114 parkin patients were similar, even for signs such as dystonia at onset and increased reflexes, which were thought to be specific to parkin. In contrast, onset in patients with PINK1 mutations was earlier and increased reflexes were found more frequently than in patients without PINK1 or parkin mutations. These results suggest that PINK1 is the second most frequent causative gene in early-onset Parkinson's disease with a slowly progressive phenotype, indistinguishable from early-onset patients with parkin mutations.
帕金森病是一种常见疾病,主要由黑质多巴胺能神经元的丧失引起。除了帕金森病基因(parkin)和DJ - 1基因的突变外,在隐性早发性帕金森病家族中还发现了PTEN诱导激酶(PINK1)基因的突变。我们在一组177个常染色体隐性帕金森病家族中筛查了parkin和PINK1突变,这些家族的发病年龄≤60岁,大多来自欧洲。在7个无亲缘关系的家族中,我们鉴定出10个致病性PINK1突变(5个错义突变、2个无义突变和3个移码缺失突变),其中8个是新发现的。所有突变均处于纯合或复合杂合状态。有趣的是,在一个有两种不同突变的家族中观察到了假显性遗传。12例PINK1患者和114例parkin患者的临床特征相似,即使是那些如发病时的肌张力障碍和反射亢进等曾被认为是parkin患者特有的体征。相比之下,PINK1突变患者的发病更早,且反射亢进的发生率比无PINK1或parkin突变的患者更高。这些结果表明,PINK1是早发性帕金森病中第二常见的致病基因,其具有缓慢进展的表型,与早发性parkin突变患者难以区分。