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肾脏发育不全/发育不良 3 型由 GREB1L 的罕见变异引起,在中国家系中表现为不完全外显。

Renal Hypodysplasia/Aplasia 3 Caused by a Rare Variant of GREB1L With Incomplete Penetrance in a Chinese Family.

机构信息

Center of Prenatal Diagnosis, Huzhou Maternity & Child Health Care Hospital, Huzhou, China.

Center of Prenatal Diagnosis, Huzhou Maternity & Child Health Care Hospital, Huzhou, China.

出版信息

Urology. 2024 Mar;185:49-53. doi: 10.1016/j.urology.2024.01.007. Epub 2024 Feb 1.

Abstract

Renal agenesis represents the most severe form of congenital anomalies of the kidney and urinary tract. Bilateral renal agenesis is almost invariably fatal at birth and has high genetic heterogeneity. Here we report on a Chinese family with two pregnancies affected by a prenatal form of bilateral renal agenesis. Trio-WES was conducted to explore the underlying genetic cause and identified a novel nonsense variant (c .2621G>A: p. Trp874Ter) in the GREB1L gene. Based on previous research, pathogenic mutations in GREB1L can cause renal hypodysplasia/aplasia-3 (RHDA3) with autosomal dominant inheritance. Sanger sequencing performed on the family members revealed that the variant was vertically transmitted from the maternal grandfather through the unaffected mother to the two affected fetuses, fully demonstrating the incomplete dominance of the disease. Our study extends the mutational spectrum associated with RHDA3 and contributes to a more general understanding for the complex genetic inheritance of GREB1L.

摘要

肾发育不全是肾脏和泌尿道先天畸形中最严重的一种。双侧肾发育不全几乎在出生时就会致命,且具有高度的遗传异质性。本研究报道了一个中国家庭的两例产前双侧肾发育不全。通过对先证者及其父母进行三代人全外显子组测序(trio-WES),发现 GREB1L 基因中存在一个新的无义变异(c.2621G>A: p. Trp874Ter)。基于既往研究,GREB1L 中的致病性突变可导致常染色体显性遗传的肾发育不全/发育不良-3 型(RHDA3)。对家系成员进行 Sanger 测序发现,该变异是从携带该变异但表型正常的外祖父垂直传递给未患病的母亲,再传递给两位患病胎儿,充分证实了该疾病的不完全外显率。本研究扩展了与 RHDA3 相关的突变谱,并为 GREB1L 复杂的遗传方式提供了更普遍的认识。

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