Department of Otolaryngology, Head and Neck Surgery, University of Iowa, Iowa City, IA 52242, USA.
Proc Natl Acad Sci U S A. 2010 Dec 7;107(49):21104-9. doi: 10.1073/pnas.1012989107. Epub 2010 Nov 15.
The extreme genetic heterogeneity of nonsyndromic hearing loss (NSHL) makes genetic diagnosis expensive and time consuming using available methods. To assess the feasibility of target-enrichment and massively parallel sequencing technologies to interrogate all exons of all genes implicated in NSHL, we tested nine patients diagnosed with hearing loss. Solid-phase (NimbleGen) or solution-based (SureSelect) sequence capture, followed by 454 or Illumina sequencing, respectively, were compared. Sequencing reads were mapped using GSMAPPER, BFAST, and BOWTIE, and pathogenic variants were identified using a custom-variant calling and annotation pipeline (ASAP) that incorporates publicly available in silico pathogenicity prediction tools (SIFT, BLOSUM, Polyphen2, and Align-GVGD). Samples included one negative control, three positive controls (one biological replicate), and six unknowns (10 samples total), in which we genotyped 605 single nucleotide polymorphisms (SNPs) by Sanger sequencing to measure sensitivity and specificity for SureSelect-Illumina and NimbleGen-454 methods at saturating sequence coverage. Causative mutations were identified in the positive controls but not in the negative control. In five of six idiopathic hearing loss patients we identified the pathogenic mutation. Massively parallel sequencing technologies provide sensitivity, specificity, and reproducibility at levels sufficient to perform genetic diagnosis of hearing loss.
常染色体显性遗传性非综合征型听力损失(DFNA)的遗传异质性很强,采用现有的方法进行基因诊断既昂贵又耗时。为了评估目标富集和大规模平行测序技术检测所有与非综合征型听力损失相关基因外显子的可行性,我们对 9 名被诊断为听力损失的患者进行了测试。分别采用固相(NimbleGen)或溶液(SureSelect)序列捕获,随后分别进行 454 或 Illumina 测序。使用 GSMAPPER、BFAST 和 BOWTIE 将测序读段映射到参考基因组,然后使用包含公共可用的计算机致病性预测工具(SIFT、BLOSUM、Polyphen2 和 Align-GVGD)的定制变异调用和注释管道(ASAP)来识别致病性变异。样本包括一个阴性对照、三个阳性对照(一个生物学重复)和六个未知样本(共 10 个样本),我们通过 Sanger 测序对 605 个单核苷酸多态性(SNP)进行了基因分型,以测量 SureSelect-Illumina 和 NimbleGen-454 方法在饱和测序覆盖度下的灵敏度和特异性。在阳性对照中发现了致病突变,但在阴性对照中没有发现。在 6 名特发性听力损失患者中的 5 名中,我们鉴定出了致病性突变。大规模平行测序技术在灵敏度、特异性和重现性方面达到了足以进行听力损失遗传诊断的水平。