DFNB8/10 家族中一种新的 TMPRSS3 错义突变阻碍了该蛋白的蛋白水解激活。
A novel TMPRSS3 missense mutation in a DFNB8/10 family prevents proteolytic activation of the protein.
作者信息
Wattenhofer Marie, Sahin-Calapoglu Nilüfer, Andreasen Ditte, Kalay Ersan, Caylan Refik, Braillard Bastien, Fowler-Jaeger Nicole, Reymond Alexandre, Rossier Bernard C, Karaguzel Ahmet, Antonarakis Stylianos E
机构信息
Department of Genetic Medicine and Development, University of Geneva Medical School, Geneva, Switzerland.
出版信息
Hum Genet. 2005 Oct;117(6):528-35. doi: 10.1007/s00439-005-1332-x. Epub 2005 Jul 14.
Pathogenic mutations in TMPRSS3, which encodes a transmembrane serine protease, cause non-syndromic deafness DFNB8/10. Missense mutations map in the low density-lipoprotein receptor A (LDLRA), scavenger-receptor cysteine-rich (SRCR), and protease domains of the protein, indicating that all domains are important for its function. TMPRSS3 undergoes proteolytic cleavage and activates the ENaC sodium channel in a Xenopus oocyte model system. To assess the importance of this gene in non-syndromic childhood or congenital deafness in Turkey, we screened for mutations affected members of 25 unrelated Turkish families. The three families with the highest LOD score for linkage to chromosome 21q22.3 were shown to harbor P404L, R216L, or Q398X mutations, suggesting that mutations in TMPRSS3 are a considerable contributor to non-syndromic deafness in the Turkish population. The mutant TMPRSS3 harboring the novel R216L missense mutation within the predicted cleavage site of the protein fails to undergo proteolytic cleavage and is unable to activate ENaC, thus providing evidence that pre-cleavage of TMPRSS3 is mandatory for normal function.
编码跨膜丝氨酸蛋白酶的TMPRSS3基因中的致病突变会导致非综合征性耳聋DFNB8/10。错义突变位于该蛋白的低密度脂蛋白受体A(LDLRA)、富含半胱氨酸的清道夫受体(SRCR)和蛋白酶结构域中,这表明所有结构域对其功能都很重要。在非洲爪蟾卵母细胞模型系统中,TMPRSS3会发生蛋白水解切割并激活ENaC钠通道。为了评估该基因在土耳其儿童非综合征性或先天性耳聋中的重要性,我们对25个不相关的土耳其家庭中受影响的成员进行了突变筛查。与21号染色体q22.3连锁的LOD得分最高的三个家庭被证明携带P404L、R216L或Q398X突变,这表明TMPRSS3基因的突变是土耳其人群非综合征性耳聋的一个重要原因。在该蛋白预测的切割位点内携带新型R216L错义突变的突变型TMPRSS3无法进行蛋白水解切割,也无法激活ENaC,因此提供了证据表明TMPRSS3的切割前状态对其正常功能是必不可少的。