Université de Tunis El Manar, Institut Pasteur de Tunis, Laboratoire de Génomique Biomédicale et Oncogénétique (LR11IPT05), BP74, 13 Place Pasteur, Belvédère, 1002 Tunis, Tunisia.
Biomed Res Int. 2013;2013:206803. doi: 10.1155/2013/206803. Epub 2013 Sep 4.
Mal de Meleda (MDM) is a rare, autosomal recessive form of palmoplantar keratoderma. It is characterized by erythema and hyperkeratosis of the palms and soles that progressively extend to the dorsal surface of the hands and feet. It is caused by mutations in SLURP-1 gene encoding for secreted mammalian Ly-6/uPAR-related protein 1 (SLURP-1). We performed mutational analysis by direct sequencing of SLURP-1 gene in order to identify the genetic defect in three unrelated families (families MDM-12, MDM-13, and MDM-14) variably affected with transgressive palmoplantar keratoderma. A spectrum of clinical presentations with variable features has been observed from the pronounced to the transparent hyperkeratosis. We identified the 82delT frame shift mutation in the SLURP-1 gene in both families MDM-12 and MDM-13 and the missense variation p.Cys99Tyr in family MDM-14. To date, the 82delT variation is the most frequent cause of MDM in the world which is in favour of a recurrent molecular defect. The p.Cys99Tyr variation is only described in Tunisian families making evidence of founder effect mutation of likely Tunisian origin. Our patients presented with very severe to relatively mild phenotypes, including multiple keratolytic pits observed for one patient in the hyperkeratotic area which was not previously reported. The phenotypic variability may reflect the influence of additional factors on disease characteristics. This report further expands the spectrum of clinical phenotypes associated with mutations in SLURP1 in the Mediterranean population.
遗传性掌跖角化过度症(MDM)是一种罕见的常染色体隐性遗传病。其特征为手掌和足底红斑和过度角化,逐渐扩展到手和脚的背侧。该病是由编码分泌型哺乳动物 Ly-6/uPAR 相关蛋白 1(SLURP-1)的 SLURP-1 基因突变引起的。我们通过直接测序 SLURP-1 基因,对三个不同家族(MDM-12、MDM-13 和 MDM-14 家族)中具有进行性掌跖角化过度症的患者进行了基因突变分析。我们观察到了一系列临床表现,从明显的过度角化到透明性角化过度,具有不同的特征。我们在 MDM-12 和 MDM-13 家族中发现了 SLURP-1 基因中的 82delT 移码突变,在 MDM-14 家族中发现了错义变异 p.Cys99Tyr。迄今为止,82delT 变异是世界上 MDM 最常见的原因,这表明存在反复出现的分子缺陷。p.Cys99Tyr 变异仅在突尼斯家族中描述,表明存在可能起源于突尼斯的创始人效应突变。我们的患者表现出非常严重到相对较轻的表型,包括一名患者在过度角化区域观察到多个角质松解性凹陷,这在以前的报道中尚未观察到。表型的可变性可能反映了其他因素对疾病特征的影响。本报告进一步扩展了地中海人群中与 SLURP1 基因突变相关的临床表型谱。