Ebermann Inga, Scholl Hendrik P N, Charbel Issa Peter, Becirovic Elvir, Lamprecht Jürgen, Jurklies Bernhard, Millán José M, Aller Elena, Mitter Diana, Bolz Hanno
Institute of Human Genetics, University Hospital of Cologne, Kerpener Str. 34, 50931 Cologne, Germany.
Hum Genet. 2007 Apr;121(2):203-11. doi: 10.1007/s00439-006-0304-0. Epub 2006 Dec 15.
Usher syndrome is an autosomal recessive condition characterized by sensorineural hearing loss, variable vestibular dysfunction, and visual impairment due to retinitis pigmentosa (RP). The seven proteins that have been identified for Usher syndrome type 1 (USH1) and type 2 (USH2) may interact in a large protein complex. In order to identify novel USH genes, we followed a candidate strategy, assuming that mutations in proteins interacting with this "USH network" may cause Usher syndrome as well. The DFNB31 gene encodes whirlin, a PDZ scaffold protein with expression in both hair cell stereocilia and retinal photoreceptor cells. Whirlin represents an excellent candidate for USH2 because it binds to Usherin (USH2A) and VLGR1b (USH2C). Genotyping of microsatellite markers specific for the DFNB31 gene locus on chromosome 9q32 was performed in a German USH2 family that had been excluded for all known USH loci. Patients showed common haplotypes. Sequence analysis of DFNB31 revealed compound heterozygosity for a nonsense mutation, p.Q103X, in exon 1, and a mutation in the splice donor site of exon 2, c.837+1G>A. DFNB31 mutations appear to be a rare cause of Usher syndrome, since no mutations were identified in an additional 96 USH2 patients. While mutations in the C-terminal half of whirlin have previously been reported in non-syndromic deafness (DFNB31), both alterations identified in our USH2 family affect the long protein isoform. We propose that mutations causing Usher syndrome are probably restricted to exons 1-6 that are specific for the long isoform and probably crucial for retinal function. We describe a novel genetic subtype for Usher syndrome, which we named USH2D and which is caused by mutations in whirlin. Moreover, this is the first case of USH2 that is allelic to non-syndromic deafness.
尤塞综合征是一种常染色体隐性疾病,其特征为感音神经性听力损失、不同程度的前庭功能障碍以及由色素性视网膜炎(RP)导致的视力损害。已确定的与1型尤塞综合征(USH1)和2型尤塞综合征(USH2)相关的七种蛋白质可能在一个大型蛋白质复合物中相互作用。为了鉴定新的尤塞综合征基因,我们采用了候选策略,假定与这个“尤塞网络”相互作用的蛋白质发生突变也可能导致尤塞综合征。DFNB31基因编码whirlin,一种PDZ支架蛋白,在毛细胞静纤毛和视网膜光感受器细胞中均有表达。Whirlin是USH2的一个极佳候选基因,因为它能与usherin(USH2A)和VLGR1b(USH2C)结合。在一个已排除所有已知尤塞综合征基因座的德国家庭中,对位于9号染色体q32上的DFNB31基因座特异性微卫星标记进行了基因分型。患者表现出常见的单倍型。DFNB31的序列分析显示,外显子1存在一个无义突变p.Q103X的复合杂合性,以及外显子2剪接供体位点的一个突变c.837+1G>A。DFNB31突变似乎是尤塞综合征的一个罕见病因,因为在另外96例USH2患者中未发现突变。虽然之前在非综合征性耳聋(DFNB31)中报道过whirlin C端的突变,但我们在USH2家系中鉴定出的这两种改变均影响长蛋白异构体。我们提出,导致尤塞综合征的突变可能局限于长异构体特有的外显子1至6,这些外显子可能对视网膜功能至关重要。我们描述了一种新的尤塞综合征遗传亚型,将其命名为USH2D,它由whirlin突变引起。此外,这是首例与非综合征性耳聋等位的USH2病例。