GM-Unit, Helsinki Institute of Life Science, University of Helsinki, Helsinki, Finland.
Stem Cells and Metabolism Research Program, Faculty of Medicine, University of Helsinki, Helsinki, Finland.
Adv Exp Med Biol. 2020;1236:109-136. doi: 10.1007/978-981-15-2389-2_5.
Congenital anomalies of the kidney and urinary tract (CAKUT) are common birth defects, which cause the majority of chronic kidney diseases in children. CAKUT covers a wide range of malformations that derive from deficiencies in embryonic kidney and lower urinary tract development, including renal aplasia, hypodysplasia, hypoplasia, ectopia, and different forms of ureter abnormalities. The majority of the genetic causes of CAKUT remain unknown. Research on mutant mice has identified multiple genes that critically regulate renal differentiation. The data generated from this research have served as an excellent resource to identify the genetic bases of human kidney defects and have led to significantly improved diagnostics. Furthermore, genetic data from human CAKUT studies have also revealed novel genes regulating kidney differentiation.
先天性肾和尿路畸形(CAKUT)是常见的出生缺陷,导致大多数儿童慢性肾脏病。CAKUT 涵盖了一系列源于胚胎肾和下尿路发育缺陷的畸形,包括肾发育不全、发育不良、发育不良、异位和不同形式的输尿管异常。大多数 CAKUT 的遗传原因仍然未知。对突变小鼠的研究已经确定了多个关键调节肾分化的基因。这些研究产生的数据已成为鉴定人类肾脏缺陷遗传基础的极好资源,并显著提高了诊断水平。此外,来自人类 CAKUT 研究的遗传数据还揭示了调节肾脏分化的新基因。