Gubler Marie-Claire, Gribouval Olivier, Morinière Vincent, Pawtowski Audrey, Antignac Corinne
INSERM, U574, Hôpital Necker Enfants Malades, 75743 Paris, France.
J Soc Biol. 2009;203(4):311-8. doi: 10.1051/jbio/2009035. Epub 2010 Feb 1.
Autosomal recessive renal tubular dysgenesis (RTD) is a clinical disorder observed in fetuses, characterized by absence or poor development of proximal tubules and early onset and persistent oligohydramnios leading to the Potter sequence, associated with skull ossification defect. The disease is uniformly severe resulting in low blood pressure and perinatal death in most cases or in chronic renal disease in the few surviving patients. Based on the phenotype and the finding of striking changes in renal renin expression (absent or massive), we hypothesized and demonstrated that genetic defects in the renin-angiotensin system (RAS) components are the underlying causes of the disease. At the present time, molecular screening has been performed in 46 families (F) and homozygous or compound heterozygous mutations have been detected in 41. They affect the genes encoding renine (9F), angiotensinogen (3F), AT1 receptor (3F) and angiotensin converting enzyme (26F). These findings highlight the importance of the RAS during human kidney development. Moreover, the identification of the disease based on precise histological and immunohistological analysis, and the research of the genetic defect, now allow genetic counseling and early prenatal diagnosis.
常染色体隐性遗传性肾小管发育不全(RTD)是一种在胎儿中观察到的临床病症,其特征为近端肾小管缺失或发育不良,以及早期出现并持续存在的羊水过少,进而导致波特序列,同时伴有颅骨骨化缺陷。该疾病通常病情严重,多数情况下会导致低血压和围产期死亡,少数存活患者则会发展为慢性肾病。基于表型以及肾素表达显著变化(缺失或大量增加)这一发现,我们推测并证实肾素 - 血管紧张素系统(RAS)组分的基因缺陷是该疾病的根本病因。目前,已对46个家系进行了分子筛查,其中41个家系检测到纯合或复合杂合突变。这些突变影响编码肾素的基因(9个家系)、血管紧张素原的基因(3个家系)、AT1受体的基因(3个家系)以及血管紧张素转换酶的基因(26个家系)。这些发现凸显了RAS在人类肾脏发育过程中的重要性。此外,基于精确的组织学和免疫组织学分析对该疾病进行诊断,以及对基因缺陷的研究,现在能够进行遗传咨询和早期产前诊断。