Mellerio J E, Salas-Alanis J C, Talamantes M L, Horn H, Tidman M J, Ashton G H, Eady R A, McGrath J A
Department of Cell and Molecular Pathology, St John's Institute of Dermatology (The Guy's, King's College and St Thomas' Hospitals' Medical and Dental School), St Thomas' Hospital, Lambeth Palace Road, London SE1 7EH, U.K.
Br J Dermatol. 1998 Oct;139(4):730-7. doi: 10.1046/j.1365-2133.1998.02496.x.
Dystrophic epidermolysis bullosa (DEB) is caused by mutations in the type VII collagen gene (COL7A1). Nearly all cases of dominant DEB are caused by glycine substitution mutations occurring within the triple helical region of type VII collagen, and most of the mutations are unique to individual families. In this study, we identified a patient of Hispanic-Mexican origin with a mild form of DEB, which resulted from a de novo dominant glycine substitution, G2043R, in exon 73 of COL7A1. We also investigated a Scottish family with a three-generation pedigree of dominant DEB, in whom the same glycine to arginine substitution mutation was demonstrated. This particular mutation has also been detected previously in three other families with dominant DEB: one Italian, one Hungarian and one Norwegian. Given the widespread geographical distribution of this mutation and the demonstration of its occurrence as a de novo event, G2043R therefore represents the first example of a mutational hotspot in dominant DEB. Interestingly, although both the Mexican and Scottish families we studied had some clinical features in keeping with the Pasini form of the disorder, there was considerable interfamilial variability as well as intrafamilial diversity in the affected individuals.
营养不良性大疱性表皮松解症(DEB)由VII型胶原蛋白基因(COL7A1)突变引起。几乎所有显性DEB病例都是由VII型胶原蛋白三螺旋区域内发生的甘氨酸替代突变导致的,并且大多数突变在各个家族中都是独特的。在本研究中,我们鉴定出一名西班牙裔墨西哥裔患者,患有轻度形式的DEB,这是由COL7A1外显子73中一个新生的显性甘氨酸替代G2043R引起的。我们还研究了一个有三代显性DEB谱系的苏格兰家族,在该家族中证实了相同的甘氨酸到精氨酸替代突变。此前在另外三个患有显性DEB的家族中也检测到了这个特定突变:一个意大利家族、一个匈牙利家族和一个挪威家族。鉴于该突变广泛的地理分布以及其作为新生事件发生的证据,G2043R因此代表了显性DEB中突变热点的首个实例。有趣的是,尽管我们研究的墨西哥和苏格兰家族都有一些与帕西尼型疾病相符的临床特征,但在受影响个体中存在相当大的家族间变异性以及家族内多样性。