Medical Genetics, University of Siena, Siena, Italy.
Gene. 2013 Sep 10;526(2):490-3. doi: 10.1016/j.gene.2013.04.070. Epub 2013 May 8.
We report on seven Iraqi patients with 46,XY karyotype and ambiguous genitalia characterized by perineo-scrotal hypospadias, bifid scrotum, clitoris like phallus, palpable testes in inguinal canal and pseudovagina. Patients were raised five as females and two as males. They are all unrelated with the exception of two couples of brothers. The diagnosis of 5-α-reductase-2 deficiency syndrome was first hypothesized on clinical grounds and then confirmed by molecular analysis. Direct sequencing analysis of the SRD5A2 gene revealed in five patients a novel homozygous frame-shift mutation (c.453delC) and in two related patients a previous reported missense mutation. The presence of the same mutation in unrelated patients of the same population suggests a possible founder effect. This report brings the 5-α-reductase-2 deficiency syndrome to the attention of clinical geneticists and child surgeons and discusses the appropriate clinical and surgical strategies for treating these patients.
我们报告了 7 例 46,XY 核型和生殖器模糊的伊拉克患者,其特征为会阴-阴囊型尿道下裂、分裂阴囊、阴蒂样阴茎、可触及的腹股沟管内睾丸和假阴道。患者中有 5 例被抚养为女性,2 例被抚养为男性。除了两对兄弟外,他们彼此之间没有关系。5-α-还原酶-2 缺乏综合征的诊断最初是基于临床依据提出的,然后通过分子分析得到证实。SRD5A2 基因的直接测序分析在 5 名患者中发现了一种新的纯合移码突变(c.453delC),在 2 名相关患者中发现了先前报道的错义突变。同一人群中无关患者存在相同的突变提示可能存在一个共同的突变来源。本报告引起了临床遗传学家和儿童外科医生对 5-α-还原酶-2 缺乏综合征的关注,并讨论了治疗这些患者的适当临床和手术策略。