Fischer J, Bouadjar B, Heilig R, Huber M, Lefèvre C, Jobard F, Macari F, Bakija-Konsuo A, Ait-Belkacem F, Weissenbach J, Lathrop M, Hohl D, Prud'homme J F
Centre National de Génotypage, 91057 Evry, France.
Hum Mol Genet. 2001 Apr 1;10(8):875-80. doi: 10.1093/hmg/10.8.875.
Mal de Meleda (MDM) is a rare autosomal recessive skin disorder, characterized by transgressive palmoplantar keratoderma (PPK), keratotic skin lesions, perioral erythema, brachydactyly and nail abnormalities. We report the refinement of our previously described interval of MDM on chromosome 8qter, and the identification of mutations in affected individuals in the ARS (component B) gene, encoding a protein named SLURP-1, for secreted Ly-6/uPAR related protein 1. This protein is a member of the Ly-6/uPAR superfamily, in which most members have been localized in a cluster on chromosome 8q24.3. The amino acid composition of SLURP-1 is homologous to that of toxins such as frog cytotoxin and snake venom neurotoxins and cardiotoxins. Three different homozygous mutations (a deletion, a nonsense and a splice site mutation) were detected in 19 families of Algerian and Croatian origin, suggesting founder effects. Moreover, one of the common haplotypes presenting the same mutation was shared by families from both populations. Secreted and receptor proteins of the Ly-6/uPAR superfamily have been implicated in transmembrane signal transduction, cell activation and cell adhesion. This is the first instance of a secreted protein being involved in a PPK.
梅勒达病(MDM)是一种罕见的常染色体隐性遗传性皮肤病,其特征为进行性掌跖角化病(PPK)、角化性皮肤损害、口周红斑、短指畸形和指甲异常。我们报告了对先前描述的位于8号染色体长臂末端的MDM致病区间的精细定位,以及在受影响个体中鉴定出ARS(组分B)基因的突变,该基因编码一种名为SLURP-1的蛋白质,即分泌型Ly-6/uPAR相关蛋白1。这种蛋白质是Ly-6/uPAR超家族的成员,该超家族的大多数成员都定位于8号染色体q24.3区域的一个基因簇中。SLURP-1的氨基酸组成与蛙细胞毒素、蛇毒神经毒素和心脏毒素等毒素的氨基酸组成同源。在19个来自阿尔及利亚和克罗地亚的家族中检测到三种不同的纯合突变(一个缺失突变、一个无义突变和一个剪接位点突变) , 提示存在奠基者效应。此外,两个群体的家族共享呈现相同突变的一种常见单倍型。Ly-6/uPAR超家族的分泌蛋白和受体蛋白与跨膜信号转导、细胞活化及细胞黏附有关。这是分泌蛋白参与掌跖角化病的首例报道。