Neurodegenerative Brain Diseases Group, Department of Molecular Genetics, VIB, Antwerpen, Belgium.
Hum Mutat. 2010 Jul;31(7):763-80. doi: 10.1002/humu.21277.
To date, molecular genetic analyses have identified over 500 distinct DNA variants in five disease genes associated with familial Parkinson disease; alpha-synuclein (SNCA), parkin (PARK2), PTEN-induced putative kinase 1 (PINK1), DJ-1 (PARK7), and Leucine-rich repeat kinase 2 (LRRK2). These genetic variants include approximately 82% simple mutations and approximately 18% copy number variations. Some mutation subtypes are likely underestimated because only few studies reported extensive mutation analyses of all five genes, by both exonic sequencing and dosage analyses. Here we present an update of all mutations published to date in the literature, systematically organized in a novel mutation database (http://www.molgen.ua.ac.be/PDmutDB). In addition, we address the biological relevance of putative pathogenic mutations. This review emphasizes the need for comprehensive genetic screening of Parkinson patients followed by an insightful study of the functional relevance of observed genetic variants. Moreover, while capturing existing data from the literature it became apparent that several of the five Parkinson genes were also contributing to the genetic etiology of other Lewy Body Diseases and Parkinson-plus syndromes, indicating that mutation screening is recommendable in these patient groups.
迄今为止,在与家族性帕金森病相关的五个疾病基因(α-突触核蛋白 (SNCA)、Parkin (PARK2)、PTEN 诱导的假定激酶 1 (PINK1)、DJ-1 (PARK7) 和富亮氨酸重复激酶 2 (LRRK2))中,分子遗传学分析已经确定了超过 500 个不同的 DNA 变异。这些遗传变异包括大约 82%的简单突变和约 18%的拷贝数变异。由于只有少数研究报告了对所有五个基因进行广泛的突变分析,包括外显子测序和剂量分析,因此某些突变亚型可能被低估了。在这里,我们展示了迄今为止在文献中发表的所有突变的最新信息,这些突变被系统地组织在一个新的突变数据库(http://www.molgen.ua.ac.be/PDmutDB)中。此外,我们还探讨了潜在致病性突变的生物学相关性。这篇综述强调了对帕金森病患者进行全面遗传筛查的必要性,然后对观察到的遗传变异的功能相关性进行深入研究。此外,在从文献中获取现有数据时,我们发现五个帕金森基因中的几个也与其他路易体疾病和帕金森综合征相关,这表明在这些患者群体中建议进行突变筛查。