The Second School of Clinical Medicine, Southern Medical University, Guangzhou 510282, Guangdong, China; Department of Pediatric Surgery, Nanhai Meternity&Child Healthcare Hospital of Foshan, Foshan 528200, Guangdong, China.
Department of Pediatric Surgery, Nanhai Meternity&Child Healthcare Hospital of Foshan, Foshan 528200, Guangdong, China.
Steroids. 2022 Aug;184:109041. doi: 10.1016/j.steroids.2022.109041. Epub 2022 May 10.
Hypospadias, a malformation of male external genitalia, is characterized by an aberrant opening of the urethra on the ventral side of the penis. It is considered a complex disorder with both environmental and genetic factors involved in its pathogenesis. To identify the genetic abnormality involved in the pathogenesis of hypospadias, we performed whole exome sequencing (WES) analysis in 42 hypospadias patients with karyotype 46, XY in the Nanhai Meternity&Child Health Hospital of Foshan. All the likely pathogenic variants were confirmed by Sanger sequencing and assessed by Sorting Intolerant from Tolerant (SIFT), PROVEAN, PolyPhen2, ClinPred, LRT, Mutation Assessor, FATHMM, and GERP software. We discovered 27 gene mutations in 20 patients, including eight cases of the SRD5A2 gene, 4 cases of the AR gene, 3 cases of the CYP17A1 gene, 1 case of the WT1 gene, 1 case of the ANOS1 gene, 1 case of the NR5A1 gene, 1 case of the FGFR1 gene, and one case of the DHX37 gene. Our study is the first to describe six novel missense mutations, AR(c.302G > A, c.2593G > T, and c.1705G > T), CYP17A1(c.1298 T > C), FGFR1 (c.995C > T) and DHX37(c.923G > A). In summary, genetic defect detection was useful for early diagnosis of severe hypospadias in the Han Chinese population. Nevertheless, most cases remain unexplained, and the exact pathogenesis of hypospadias still needs further study.
尿道下裂是一种男性外生殖器的畸形,其特征是尿道在阴茎腹侧异常开口。它被认为是一种复杂的疾病,其发病机制涉及环境和遗传因素。为了确定参与尿道下裂发病机制的遗传异常,我们对在佛山市南海区妇幼保健院就诊的 42 名核型为 46,XY 的尿道下裂患者进行了全外显子组测序(WES)分析。通过 Sanger 测序对所有可能的致病性变异进行了确认,并通过 Sorting Intolerant from Tolerant(SIFT)、PROVEAN、PolyPhen2、ClinPred、LRT、Mutation Assessor、FATHMM 和 GERP 软件进行了评估。我们在 20 名患者中发现了 27 个基因突变,包括 8 例 SRD5A2 基因、4 例 AR 基因、3 例 CYP17A1 基因、1 例 WT1 基因、1 例 ANOS1 基因、1 例 NR5A1 基因、1 例 FGFR1 基因和 1 例 DHX37 基因。我们的研究首次描述了 6 种新的错义突变,AR(c.302G>A、c.2593G>T 和 c.1705G>T)、CYP17A1(c.1298T>C)、FGFR1(c.995C>T)和 DHX37(c.923G>A)。总之,遗传缺陷检测有助于对汉族重度尿道下裂患者进行早期诊断。然而,大多数病例仍无法解释,尿道下裂的确切发病机制仍需要进一步研究。