Ozer Esra Arun, Aksu Nejat, Erdogan Hakan, Yavascan Onder, Kara Orhan, Gribouval Olivier, Gubler Marie-Claire, Antignac Corinne
Sosyal Sigortalar Kurumu Tepecik Teaching Hospital, Clinics of Pediatrics, Yenisehir Izmir, Turkey.
Nephrology (Carlton). 2004 Oct;9(5):310-2. doi: 10.1111/j.1440-1797.2004.00324.x.
We report in this paper two siblings aged 8 and 17 months who were clinically diagnosed with familial steroid-resistant nephrotic syndrome (SRNS). By mutation screening of the NPHS2 gene, a homozygous missense mutation, P118L, was detected in both children. This study is the first systematic investigation of NPHS2 gene mutations in Turkish children with familial SRNS. If this mutation is a hot spot of mutation in the Turkish population, screening this novel mutation in Turkish children with SRNS may be of great clinical use to prevent unnecessary treatment modalities, provide accurate genetic counselling and predict the prognosis of the disease.
我们在本文中报告了两名分别为8个月和17个月大的兄弟姐妹,他们临床诊断为家族性类固醇抵抗性肾病综合征(SRNS)。通过对NPHS2基因进行突变筛查,在两名儿童中均检测到纯合错义突变P118L。本研究是对土耳其患有家族性SRNS儿童的NPHS2基因突变进行的首次系统调查。如果该突变是土耳其人群中的突变热点,那么对土耳其患有SRNS的儿童筛查这种新突变可能在预防不必要的治疗方式、提供准确的遗传咨询以及预测疾病预后方面具有重大临床意义。