Rooryck Caroline, Roudaut Christel, Robine Eulalie, Müsebeck Jörg, Arveiler Benoît
Service de Génétique Médicale, Centre Hospitalier Universitaire, Bordeaux, France.
Pigment Cell Res. 2006 Jun;19(3):239-42. doi: 10.1111/j.1600-0749.2006.00298.x.
Non-syndromic oculocutaneous albinism (OCA) is a clinically and genetically heterogeneous autosomal recessive disorder with mutations identified in several genes: OCA1 (tyrosinase, TYR), OCA2 (OCA2), OCA3 (tyrosinase-related protein 1, TYRP1), and OCA4 (membrane-associated transporter protein, MATP). OCA3 was thought to be restricted to black populations, where it was clinically described as rufous or brown albinism, until the recent report of a homozygous TYRP1 mutation in Caucasian patients from a consanguineous Pakistani family. Here, we describe a German patient of Caucasian origin, with a light-yellow skin, yellow-gold hair with orange highlights, fair eyelashes, several pigmented naevi, and no tendency to tan, only to burn. Eye-colour is blue-green with substance defects of the iris. Molecular analysis did not reveal any mutation in the TYR and OCA2 genes. Two mutations were found in the TYRP1 gene: a missense mutation (c.1066G>A/p.Arg356Glu) that was inherited from the mother, and a de novo single-base deletion (c.106delT/p.Leu36X). This finding suggests that mutation screening should be extended to the TYRP1 gene in patients from all ethnic origins, at least in cases where no mutations have been identified in the other OCA genes.
非综合征性眼皮肤白化病(OCA)是一种临床和遗传异质性的常染色体隐性疾病,已在多个基因中发现突变:OCA1(酪氨酸酶,TYR)、OCA2(OCA2)、OCA3(酪氨酸酶相关蛋白1,TYRP1)和OCA4(膜相关转运蛋白,MATP)。OCA3曾被认为仅限于黑人人群,在临床上被描述为红棕色或棕色白化病,直到最近有报道称,来自一个巴基斯坦近亲家庭的白种人患者存在纯合TYRP1突变。在此,我们描述了一名德国白种人患者,其皮肤呈浅黄色,头发为金黄带橙色亮点,睫毛浅色,有多个色素痣,无晒黑倾向,只有晒伤倾向。眼睛颜色为蓝绿色,虹膜有实质性缺陷。分子分析未在TYR和OCA2基因中发现任何突变。在TYRP1基因中发现了两个突变:一个错义突变(c.1066G>A/p.Arg356Glu),从母亲遗传而来,以及一个新生的单碱基缺失(c.106delT/p.Leu36X)。这一发现表明,至少在其他OCA基因未发现突变的情况下,对所有种族的患者进行突变筛查时应将范围扩大到TYRP1基因。