Shemanko C S, Mellerio J E, Tidman M J, Lane E B, Eady R A
Department of Anatomy and Physiology, University of Dundee, UK.
J Invest Dermatol. 1998 Nov;111(5):893-5. doi: 10.1046/j.1523-1747.1998.00388.x.
Mutant keratins 5 or 14 are implicated in the etiology of epidermolysis bullosa simplex (EBS). The catalog of mutations has established certain patterns of mutation clusters from which it may be possible, along with associated biochemical data, to predict phenotypic severity. It is becoming apparent that some of these assumptions may now require modification. We report a mutation in the gene encoding keratin 14 (KRT14) that changes the predicted amino acid at position 119, at the start of the helix initiation motif, from methionine to threonine (K14 M119T) in a patient with an EBS Dowling-Meara phenotype with severe palmo-plantar hyperkeratosis. This demonstrates that the three major types of EBS can arise from missense mutations in the same codon. The findings suggest that the specific nature of the missense mutation, in the context of the protein sequence, can contribute far more to the clinical severity than previously thought. The different EBS subtypes should be viewed as gradations of clinical severity rather than distinct genetic diseases.
突变的角蛋白5或14与单纯性大疱性表皮松解症(EBS)的病因有关。突变目录已经确定了某些突变簇模式,结合相关生化数据,从中有可能预测表型严重程度。现在越来越明显的是,其中一些假设可能需要修正。我们报告了一例患有伴有严重掌跖角化过度的EBS Dowling-Meara表型患者,其编码角蛋白14(KRT14)的基因发生突变,该突变使螺旋起始基序起始处第119位的预测氨基酸从甲硫氨酸变为苏氨酸(K14 M119T)。这表明三种主要类型的EBS可由同一密码子中的错义突变引起。研究结果表明,在蛋白质序列背景下,错义突变的具体性质对临床严重程度的影响可能比以前认为的要大得多。不同的EBS亚型应被视为临床严重程度的不同等级,而不是不同的遗传疾病。