Schimmenti L A, Pierpont M E, Carpenter B L, Kashtan C E, Johnson M R, Dobyns W B
Department of Pediatrics, University of Minnesota School of Medicine, Minneapolis, USA.
Am J Med Genet. 1995 Nov 6;59(2):204-8. doi: 10.1002/ajmg.1320590217.
We describe a father and 3 sons with optic nerve colobomas, vesicoureteral reflux, and renal anomalies. The youngest son had congenital renal failure and ultimately underwent renal transplantation. The father and one son had high frequency hearing loss. There were no other affected relatives. We conclude that the association of optic nerve colobomas, renal anomalies, and vesicoureteral reflux comprises a unique autosomal dominant syndrome. Molecular investigations have determined this disorder to be associated with a single nucleotide deletion in the PAX2 gene.
我们描述了一位父亲和三个儿子,他们患有视神经缺损、膀胱输尿管反流和肾脏异常。最小的儿子患有先天性肾衰竭,最终接受了肾移植。父亲和一个儿子有高频听力损失。没有其他受影响的亲属。我们得出结论,视神经缺损、肾脏异常和膀胱输尿管反流的关联构成一种独特的常染色体显性综合征。分子研究已确定这种疾病与PAX2基因中的单个核苷酸缺失有关。