Kienzler A-K, Hargreaves C E, Patel S Y
NIHR Oxford Biomedical Research Centre, Clinical Immunology Group, Oxford, UK.
Clin Exp Immunol. 2017 Jun;188(3):326-332. doi: 10.1111/cei.12947. Epub 2017 Mar 29.
The advent of next-generation sequencing (NGS) and 'omic' technologies has revolutionized the field of genetics, and its implementation in health care has the potential to realize precision medicine. Primary immunodeficiencies (PID) are a group of rare diseases which have benefited from NGS, with a massive increase in causative genes identified in the past few years. Common variable immunodeficiency disorders (CVID) are a heterogeneous form of PID and the most common form of antibody failure in children and adults. While a monogenic cause of disease has been identified in a small subset of CVID patients, a genomewide association study and whole genome sequencing have found that, in the majority, a polygenic cause is likely. Other NGS technologies such as RNA sequencing and epigenetic studies have contributed further to our understanding of the contribution of altered gene expression in CVID pathogenesis. We believe that to unravel further the complexities of CVID, a multi-omic approach, combining DNA sequencing with gene expression, methylation, proteomic and metabolomics data, will be essential to identify novel disease-associated pathways and therapeutic targets.
新一代测序(NGS)和“组学”技术的出现彻底改变了遗传学领域,其在医疗保健中的应用有可能实现精准医学。原发性免疫缺陷病(PID)是一类罕见疾病,受益于NGS技术,在过去几年中,已鉴定出的致病基因大幅增加。常见变异型免疫缺陷病(CVID)是PID的一种异质性形式,是儿童和成人中最常见的抗体缺陷形式。虽然在一小部分CVID患者中已确定了单基因致病原因,但全基因组关联研究和全基因组测序发现,大多数情况下可能是多基因致病。其他NGS技术,如RNA测序和表观遗传学研究,进一步有助于我们理解基因表达改变在CVID发病机制中的作用。我们认为,要进一步揭示CVID的复杂性,将DNA测序与基因表达、甲基化、蛋白质组学和代谢组学数据相结合的多组学方法对于识别新的疾病相关途径和治疗靶点至关重要。