Unidade de Endocrinologia do Desenvolvimento, Laboratório de Hormônios e Genética Molecular/LIM42, Hospital das Clínicas, Disciplina de Endocrinologia, do Departamento de Clínica Médica, Faculdade de Medicina da Universidade de São Paulo, São Paulo, Brazil.
Endocrine Oncology Unit, Instituto do Câncer do Estado de São Paulo, ICESP, Faculdade de Medicina da Universidade de São Paulo, São Paulo, Brazil.
Sex Dev. 2022;16(2-3):171-183. doi: 10.1159/000525119. Epub 2022 Jul 6.
The 5α-reductase type 2 enzyme catalyzes the conversion of testosterone into dihydrotestosterone, playing a crucial role in male development. This enzyme is encoded by the SRD5A2 gene, which maps to chromosome 2 (2p23), consists of 5 exons and 4 introns, and encodes a 254 amino acid protein. Disruptions in this gene are the molecular etiology of a subgroup of differences of sex development (DSD) in 46,XY patients. Affected individuals present a large range of external genitalia undervirilization, ranging from almost typically female external genitalia to predominantly typically male external genitalia with minimal undervirilization, including isolated micropenis. This is an updated review of the implication of the SRD5A2 gene in 5α-reductase type 2 enzyme deficiency. For that, we identified 451 cases from 48 countries of this particular 46,XY DSD from the literature with reported variants in the SRD5A2 gene. Herein, we present the SRD5A2 mutational profile, the SRD5A2 polymorphisms, and the functional studies related to SRD5A2 variants to detail the molecular etiology of this condition.
5α-还原酶 2 型酶催化睾酮转化为二氢睾酮,在男性发育中起着关键作用。该酶由 SRD5A2 基因编码,该基因位于染色体 2(2p23)上,由 5 个外显子和 4 个内含子组成,编码 254 个氨基酸的蛋白质。该基因的突变是 46,XY 患者中一部分性别发育差异(DSD)的分子病因。受影响的个体表现出广泛的外生殖器未发育,从几乎典型的女性外生殖器到主要为典型的男性外生殖器,仅有轻微的未发育,包括孤立性小阴茎。这是对 SRD5A2 基因在 5α-还原酶 2 型酶缺乏症中的作用的更新综述。为此,我们从文献中确定了 48 个国家的 451 例 46,XY DSD 病例,这些病例均报道了 SRD5A2 基因中的变异。在此,我们介绍了 SRD5A2 的突变特征、SRD5A2 多态性以及与 SRD5A2 变异相关的功能研究,以详细阐述该疾病的分子病因。