Schlaubitz Silke, Yatsenko Svetlana A, Smith Laurie D, Keller Kory L, Vissers Lisenka E, Scott Daryl A, Cai Wei Wen, Reardon William, Abdul-Rahman Omar A, Lammer Edward J, Lifchez Caroline A, Magenis Ellen, Veltman Joris A, Stankiewicz Pawel, Zabel Bernhard U, Lee Brendan
Department of Molecular and Human Genetics, Baylor College of Medicine, Houston, Texas 77030, USA.
Am J Med Genet A. 2007 May 15;143A(10):1071-81. doi: 10.1002/ajmg.a.31685.
We describe our findings in a 46,XY female with a clinical features of Genitopatellar syndrome (GPS) and confirmed hermaphroditism with ovotestes, and five additional patients with GPS. GPS is a genetic disorder characterized by renal and genital anomalies, joint dislocation, aplastic or hypoplastic and often displaced patellae, minor facial anomalies, and mental retardation. The genital anomalies clearly distinguish GPS from nail-patella syndrome (NPS) that has similar features, but additionally shows hypoplastic finger- and toenails as found in the 46,XY female. In our patients no mutation was found in the coding regions of WNT4, WNT7A, TBX4, and LMX1B. Fluorescent in situ hybridization (FISH) and array-based comparative genome hybridization (aCGH) analysis showed a 3 Mb deletion of LMX1B, NR6A1, and NR5A1 (SF1) in the 46,XY female. This is the first report of a microdeletion causing haploinsuffiency of LMX1B and NR5A1. The deletion of LMX1B is responsible for the knee anomalies and the deletion of NR5A1 likely causes the sex reversal. Cytogenetic analysis of the five additional patients with diagnosed GPS failed to identify a similar microdeletion, or inversion of a potentially regulatory element between the two genes. This suggests that the locus 9q33-9q34 can be excluded for GPS and that the presented case is unique in its combination of GPS and NPS features caused by a microdeletion associated with loss of function of LMX1B and NR5A1.
我们描述了一名具有生殖髌综合征(GPS)临床特征且经证实为具有卵睾的雌雄同体的46,XY女性患者的研究结果,以及另外五名GPS患者。GPS是一种遗传性疾病,其特征为肾脏和生殖器异常、关节脱位、髌骨发育不全或发育不良且常移位、轻微面部异常以及智力迟钝。生殖器异常明显将GPS与具有相似特征的指甲髌骨综合征(NPS)区分开来,但46,XY女性患者还表现出指(趾)甲发育不全,这在NPS中也有发现。在我们的患者中,未在WNT4、WNT7A、TBX4和LMX1B的编码区域发现突变。荧光原位杂交(FISH)和基于阵列的比较基因组杂交(aCGH)分析显示,该46,XY女性患者存在LMX1B、NR6A1和NR5A1(SF1)的3 Mb缺失。这是关于导致LMX1B和NR5A1单倍体不足的微缺失的首次报告。LMX1B的缺失导致膝盖异常,而NR5A1的缺失可能导致性反转。对另外五名确诊为GPS的患者进行的细胞遗传学分析未能识别出类似的微缺失,或两个基因之间潜在调控元件的倒位。这表明9q33 - 9q34位点可排除在GPS之外,并且所呈现的病例在由与LMX1B和NR5A1功能丧失相关的微缺失导致的GPS和NPS特征组合方面是独特的。