Clark Lorraine N, Louis Elan D
Department of Pathology and Cell Biology, College of Physicians and Surgeons, and Taub Institute for Research on Alzheimer's Disease and the Aging Brain, Columbia University, New York, NY, United States.
Taub Institute for Research on Alzheimer's Disease and the Aging Brain, Columbia University, New York; and Departments of Neurology and of Chronic Disease Epidemiology and Center for Neuroepidemiology and Clinical Neurological Research, Yale School of Medicine, New Haven, CT, United States.
Handb Clin Neurol. 2018;147:229-239. doi: 10.1016/B978-0-444-63233-3.00015-4.
Essential tremor (ET) is one of the most common neurologic disorders, and genetic factors are thought to contribute significantly to disease etiology. There has been a relative lack of progress in understanding the genetic etiology of ET. This could reflect a number of factors, including the presence of substantial phenotypic and genotypic heterogeneity. Thus, a meticulous approach to phenotyping is important for genetic research. A lack of standardized phenotyping across studies and patient centers likely has contributed to the relative lack of success of genomewide association studies in ET. To dissect the genetic architecture of ET, whole-genome sequencing will likely be of value. This will allow specific hypotheses about the mode of inheritance and genetic architecture to be tested. A number of approaches still remain unexplored in ET genetics, including the contribution of copy number variants, uncommon moderate-effect alleles, rare variant large-effect alleles (including Mendelian and complex/polygenic modes of inheritance), de novo and gonadal mosaicism, epigenetic changes, and noncoding variation.
特发性震颤(ET)是最常见的神经系统疾病之一,遗传因素被认为在疾病病因中起着重要作用。在理解ET的遗传病因方面进展相对缓慢。这可能反映了多种因素,包括存在大量的表型和基因型异质性。因此,细致的表型分析方法对遗传研究很重要。不同研究和患者中心缺乏标准化的表型分析可能导致了ET全基因组关联研究相对缺乏成功。为了剖析ET的遗传结构,全基因组测序可能会有价值。这将允许对遗传模式和遗传结构的特定假设进行检验。ET遗传学中仍有许多方法未被探索,包括拷贝数变异、不常见的中等效应等位基因、罕见的大效应等位基因(包括孟德尔遗传和复杂/多基因遗传模式)、新发突变和生殖腺嵌合体、表观遗传变化以及非编码变异的作用。