Department of Medicine, University of Colorado Denver, 12700 East 19th Avenue, 8611, Aurora, CO 80045, USA.
Am J Respir Crit Care Med. 2012 Dec 1;186(11):1087-94. doi: 10.1164/rccm.201207-1178PP. Epub 2012 Aug 30.
Common genetic risk variants identified by genome-wide association studies have explained a small portion of disease heritability in complex diseases. It is becoming apparent that each gene/locus is heterogeneous and that multiple rare independent risk alleles across the population contribute to disease risk. Next-generation sequencing technologies have reached the maturity and low cost necessary to perform whole genome, whole exome, and targeted region sequencing to identify all rare risk alleles across a population, a task that is not possible to achieve by genotyping. Design of whole genome, whole exome, and targeted sequencing projects to identify disease variants for complex lung diseases requires four main steps: library preparation, sequencing, sequence data analysis, and statistical analysis. Although data analysis approaches are still evolving, a number of published studies have successfully identified rare variants associated with complex disease. Despite many challenges that lie ahead in applying these technologies to lung disease, rare variants are likely to be a critical piece of the puzzle that needs to be solved to understand the genetic basis of complex lung disease and to use this information to develop better therapies.
常见的全基因组关联研究鉴定的遗传风险变异仅能解释复杂疾病部分的遗传度。越来越明显的是,每个基因/位点都是异质的,人群中多个罕见的独立风险等位基因会导致疾病风险。新一代测序技术已经达到了成熟和低成本的必要条件,可进行全基因组、全外显子和靶向区域测序,以鉴定人群中所有罕见的风险等位基因,这是基因分型无法实现的任务。设计用于鉴定复杂肺部疾病的全基因组、全外显子和靶向测序项目需要四个主要步骤:文库制备、测序、序列数据分析和统计分析。尽管数据分析方法仍在不断发展,但许多已发表的研究已经成功鉴定了与复杂疾病相关的罕见变异。尽管在将这些技术应用于肺部疾病方面还面临许多挑战,但罕见变异很可能是解决复杂肺部疾病遗传基础并利用这些信息开发更好治疗方法这一难题的关键部分。