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一个新的白蛋白基因突变导致一名年轻土耳其女性出现无白蛋白血症。

A novel frameshift deletion in the albumin gene causes analbuminemia in a young Turkish woman.

机构信息

Laboratory on Pathophysiology of Uremia, Istituto Giannina Gaslini IRCCS, Largo G. Gaslini 5, 16148 Genova, Italy.

出版信息

Clin Chim Acta. 2010 Nov 11;411(21-22):1711-5. doi: 10.1016/j.cca.2010.07.009. Epub 2010 Jul 16.

Abstract

BACKGROUND

Analbuminemia is a rare autosomal recessive disorder manifested by the absence, or severe reduction, of circulating serum albumin. The analbuminemic trait was diagnosed in a young Turkish woman on the basis of her clinical symptoms (bilateral lower limb edema) and biochemical findings (minimal albumin amount and variable increases in other protein fractions).

METHODS

Total DNA from the analbuminemic proband and her parents was PCR-amplified using oligonucleotide primers designed to amplify the 14 exons of the albumin gene (ALB) and the flanking intron regions. The products were screened for mutations by single-strand conformation polymorphism (SSCP) and heteroduplex analyses (HA).

RESULTS

HA allowed the identification of the mutation site in exon 12. Direct DNA sequencing of this abnormal fragment revealed that the analbuminemic trait was caused by a homozygous CA deletion at nucleotide positions c. 1614-1615 in the codons for Cys538 and Thr539. The subsequent frameshift should give rise to a putative truncated albumin variant in which the sequence Cys(538)-Thr-Leu-Ser has been changed to Cys(538)-Thr-Phe-Stop. The parents were heterozygous for the same mutation.

CONCLUSIONS

Gel-based mutation detection and DNA sequencing substantiate the clinical diagnosis of congenital analbuminemia in our patient and show that the condition is caused by a novel mutation within the ALB gene. These results contribute to shed light on the molecular basis of this rare condition.

摘要

背景

白蛋白血症是一种罕见的常染色体隐性遗传疾病,表现为循环血清白蛋白缺失或严重减少。根据一名年轻土耳其女性的临床症状(双侧下肢水肿)和生化发现(白蛋白量极小,其他蛋白部分增加不定),诊断出该女性患有白蛋白血症。

方法

用设计的寡核苷酸引物对白蛋白基因(ALB)的 14 个外显子和侧翼内含子区域进行聚合酶链反应(PCR)扩增分析先证者及其父母的总 DNA。用单链构象多态性(SSCP)和异源双链分析(HA)筛选突变。

结果

HA 确定了 12 号外显子的突变部位。对这一异常片段进行直接 DNA 测序,发现白蛋白血症是由于 Cys538 和 Thr539 密码子的核苷酸位置 c.1614-1615 处的 CA 缺失引起的纯合子。随后的移码应导致假定的截断白蛋白变异体,其中序列 Cys(538)-Thr-Leu-Ser 已变为 Cys(538)-Thr-Phe-Stop。父母为同一突变的杂合子。

结论

凝胶基突变检测和 DNA 测序证实了我们患者先天性白蛋白血症的临床诊断,并表明该病症是由 ALB 基因内的一个新突变引起的。这些结果有助于阐明这种罕见疾病的分子基础。

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