Smith Moyra
Genetics and Genomic Medicine, Pediatrics, School of Medicine, University of CaliforniaIrvine, CA, USA.
Front Mol Biosci. 2017 May 3;4:24. doi: 10.3389/fmolb.2017.00024. eCollection 2017.
Delineation of underlying genomic and genetic factors in a specific disease may be valuable in establishing a definitive diagnosis and may guide patient management and counseling. In addition, genetic information may be useful in identification of at risk family members. Gene mapping and initial genome sequencing data enabled the development of microarrays to analyze genomic variants. The goal of this review is to consider different generations of sequencing techniques and their application to exome sequencing and whole genome sequencing and their clinical applications. In recent decades, exome sequencing has primarily been used in patient studies. Discussed in some detail, are important measures that have been developed to standardize variant calling and to assess pathogenicity of variants. Examples of cases where exome sequencing has facilitated diagnosis and led to improved medical management are presented. Whole genome sequencing and its clinical relevance are presented particularly in the context of analysis of nucleotide and structural genomic variants in large population studies and in certain patient cohorts. Applications involving analysis of cell free DNA in maternal blood for prenatal diagnosis of specific autosomal trisomies are reviewed. Applications of DNA sequencing to diagnosis and therapeutics of cancer are presented. Also discussed are important recent diagnostic applications of DNA sequencing in cancer, including analysis of tumor derived cell free DNA and exosomes that are present in body fluids. Insights gained into underlying pathogenetic mechanisms of certain complex common diseases, including schizophrenia, macular degeneration, neurodegenerative disease are presented. The relevance of different types of variants, rare, uncommon, and common to disease pathogenesis, and the continuum of causality, are addressed. Pharmogenetic variants detected by DNA sequence analysis are gaining in importance and are particularly relevant to personalized and precision medicine.
明确特定疾病潜在的基因组和遗传因素,对于确立明确诊断、指导患者管理及咨询可能具有重要价值。此外,遗传信息有助于识别有患病风险的家庭成员。基因定位和初始基因组测序数据推动了用于分析基因组变异的微阵列技术的发展。本综述的目的是探讨不同代次的测序技术及其在外显子组测序和全基因组测序中的应用以及它们的临床应用。近几十年来,外显子组测序主要用于患者研究。本文详细讨论了为标准化变异检测及评估变异致病性而制定的重要措施。文中列举了外显子组测序有助于诊断并改善医疗管理的病例。全基因组测序及其临床相关性,特别是在大型人群研究和某些患者队列中对核苷酸和基因组结构变异分析的背景下进行了阐述。还综述了涉及分析母血中游离DNA以进行特定常染色体三体产前诊断的应用。介绍了DNA测序在癌症诊断和治疗中的应用。同时也讨论了DNA测序在癌症中近期的重要诊断应用,包括分析体液中存在的肿瘤来源游离DNA和外泌体。文中还介绍了对某些复杂常见疾病(包括精神分裂症、黄斑变性、神经退行性疾病)潜在致病机制的认识。探讨了不同类型变异(罕见、不常见和常见)与疾病发病机制的相关性以及因果关系的连续性。通过DNA序列分析检测到的药物遗传学变异正变得越来越重要,尤其与个性化和精准医学相关。