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X 连锁显性遗传性 Alport 综合征男性患者中胶原 IVα5 链阳性的临床表现较轻。

Milder clinical aspects of X-linked Alport syndrome in men positive for the collagen IV α5 chain.

机构信息

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

Department of Pediatrics, Wakayama Medical University, Wakayama, Japan.

出版信息

Kidney Int. 2014 May;85(5):1208-13. doi: 10.1038/ki.2013.479. Epub 2013 Dec 4.

Abstract

X-linked Alport syndrome is caused by mutations in the COL4A5 gene encoding the type IV collagen α5 chain (α5(IV)). Complete absence of α5(IV) in the renal basal membrane is considered a pathological characteristic in male patients; however, positive α5(IV) staining has been found in over 20% of patients. We retrospectively studied 52 genetically diagnosed male X-linked Alport syndrome patients to evaluate differences in clinical characteristics and renal outcomes between 15 α5(IV)-positive and 37 α5(IV)-negative patients. Thirteen patients in the α5(IV)-positive group had non-truncating mutations consisting of nine missense mutations, three in-frame deletions, and one splice-site mutation resulting in small in-frame deletions of transcripts. The remaining two showed somatic mutations with mosaicism. Missense mutations in the α5(IV)-positive group were more likely to be located before exon 25 compared with missense mutations in the α5(IV)-negative group. Furthermore, urinary protein levels were significantly lower and the age at onset of end-stage renal disease was significantly higher in the positive group than in the negative group. These results help to clarify the milder clinical manifestations and molecular characteristics of male X-linked Alport syndrome patients expressing the α5(IV) chain.

摘要

X 连锁显性遗传性肾炎是由编码 IV 型胶原α5 链(α5(IV))的 COL4A5 基因突变引起的。男性患者的肾脏基底膜中完全缺乏α5(IV)被认为是一种病理特征;然而,已有超过 20%的患者发现α5(IV)染色阳性。我们回顾性研究了 52 例经基因诊断的男性 X 连锁显性遗传性肾炎患者,以评估 15 例α5(IV)阳性和 37 例α5(IV)阴性患者之间的临床特征和肾脏结局差异。α5(IV)阳性组中有 13 例患者存在非截断突变,包括 9 个错义突变、3 个框内缺失和 1 个剪接位点突变,导致转录物的小框内缺失。另外两个表现出嵌合体的体细胞突变。与α5(IV)阴性组相比,α5(IV)阳性组的错义突变更可能位于外显子 25 之前。此外,阳性组的尿蛋白水平显著较低,终末期肾病的发病年龄显著较高。这些结果有助于阐明表达α5(IV)链的男性 X 连锁显性遗传性肾炎患者更轻微的临床表现和分子特征。

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