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在有20年蛋白尿病史的三联体中鉴定CUBN变体。

Identification of CUBN variants in triplets with a 20-year history of proteinuria.

作者信息

Yamamura-Miyazaki Natsumi, Sakakibara Nana, Nozu Kandai, Shima Yuko, Satomura Kenichi, Yamamoto Satoko, Baba Minato, Fujiwara Kaori, Yamamoto Katsusuke, Michigami Toshimi

机构信息

Department of Pediatric Nephrology and Metabolism, Osaka Women's and Children's Hospital, 840 Murodo-Cho, Izumi, Osaka, 594-1101, Japan.

Department of Pediatrics, Kobe University Graduate School of Medicine, Hyogo, Japan.

出版信息

CEN Case Rep. 2025 Apr;14(2):145-150. doi: 10.1007/s13730-024-00919-6. Epub 2024 Aug 5.

Abstract

CUBN encodes cubilin, which plays a role in the reabsorption of glomerular-filtered albumin in the proximal tubule. CUBN-related proteinuria was recently established as a new disease concept and may be present in proteinuric cases that were previously undiagnosed either genetically or histologically. We herein report a case of triplets diagnosed with chronic benign proteinuria due to CUBN variants 20 years after its onset. The proband, the first child of triplets, tested positive for urinary protein several times during the neonatal period. A urine screening test at 3 years old was positive. Proteinuria persisted for years within a non-nephrotic range. Kidney biopsy at 8 years old revealed minor glomerular abnormalities. Renin-angiotensin system inhibitors were started for albumin-based proteinuria but were ineffective. Since the two other triplets had similar courses, analyses of the NPHS1/2 and WT1 genes were performed but revealed no abnormalities. The triplets transitioned to adult care at 15 years old. CUBN-related proteinuria was reported in 2020; therefore, we re-analyzed their DNA samples and identified compound heterozygous variants in CUBN in all three triplets. The molecular diagnosis of CUBN-related proteinuria will save patients from unnecessary treatments and concerns about renal prognosis.

摘要

CUBN基因编码cubilin,其在近端小管中对肾小球滤过的白蛋白重吸收起作用。CUBN相关蛋白尿最近被确立为一种新的疾病概念,可能存在于既往在基因或组织学上未被诊断的蛋白尿病例中。我们在此报告一例三胞胎病例,在发病20年后被诊断为因CUBN变异导致的慢性良性蛋白尿。先证者是三胞胎中的老大,在新生儿期多次尿蛋白检测呈阳性。3岁时的尿液筛查试验呈阳性。蛋白尿在非肾病范围内持续多年。8岁时的肾活检显示轻度肾小球异常。因基于白蛋白的蛋白尿开始使用肾素 - 血管紧张素系统抑制剂,但无效。由于另外两个三胞胎有相似病程,对NPHS1/2和WT1基因进行了分析,但未发现异常。三胞胎在15岁时转至成人护理。2020年报道了CUBN相关蛋白尿;因此,我们重新分析了他们的DNA样本,在所有三个三胞胎中均鉴定出CUBN的复合杂合变异。CUBN相关蛋白尿的分子诊断将使患者免于不必要的治疗以及对肾脏预后的担忧。

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Proximal Tubules Have the Capacity to Regulate Uptake of Albumin.近端小管具有调节白蛋白摄取的能力。
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