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最常见的 DCLRE1C(ARTEMIS)突变基于同源重组事件。

The most frequent DCLRE1C (ARTEMIS) mutations are based on homologous recombination events.

机构信息

Institute for Transfusion Medicine, University of Ulm, Ulm, Germany.

出版信息

Hum Mutat. 2010 Feb;31(2):197-207. doi: 10.1002/humu.21168.

Abstract

The nuclease ARTEMIS is an essential factor of V(D)J recombination during lymphocyte development and in the repair of DNA double-strand breaks (DSB) by the nonhomologous end joining (NHEJ) pathway. Patients with mutations in the DCLRE1C gene, which encodes ARTEMIS, suffer from radiosensitive B(-/low) T(-/low) severe combined immunodeficiency (SCID) or radiosensitive Omenn syndrome. To date, causative DCLRE1C mutations inherited as a recessive trait have been reported in 49 patients. In this study, molecular diagnoses of 29 novel patients presenting with the phenotype of B(-/low) SCID revealed mutations in the DCLRE1C gene. In total, 13 different mutated DCLRE1C alleles were detected, nine of which have not been described before. By far the most frequent mutations (59%) were gross deletions of exons 1-3 or exons 1-4 due to a homologous recombination of the wild-type DCLRE1C gene with a pseudo-DCLRE1C gene located 61.2 kb 5' to the DCLRE1C start codon. Fine mapping of the recombination intervals revealed private mutations in most cases. MEIG1, a gene encoding a protein that is essential for spermatogenesis in mice, is lost by the gross deletions. Functional analyses on patients' fibroblasts demonstrated that the corresponding alleles carry null mutations of the DCLRE1C gene.

摘要

核酸酶 ARTEMIS 是淋巴细胞发育过程中 V(D)J 重组和非同源末端连接(NHEJ)途径修复 DNA 双链断裂(DSB)的一个重要因素。编码 ARTEMIS 的 DCLRE1C 基因突变的患者患有辐射敏感的 B(-/低) T(-/低)严重联合免疫缺陷(SCID)或辐射敏感的 Omenn 综合征。迄今为止,已在 49 名患者中报告了作为隐性遗传特征继承的致病 DCLRE1C 突变。在这项研究中,对 29 名表现出 B(-/低) SCID 表型的新患者进行了分子诊断,发现了 DCLRE1C 基因的突变。总共检测到 13 个不同的突变 DCLRE1C 等位基因,其中 9 个以前未描述过。迄今为止最常见的突变(59%)是由于野生型 DCLRE1C 基因与位于 DCLRE1C 起始密码子 5'端 61.2kb 处的假 DCLRE1C 基因之间的同源重组导致的外显子 1-3 或外显子 1-4 的大片段缺失。重组间隔的精细作图显示,在大多数情况下存在个体突变。MEIG1 是一种编码在小鼠中对精子发生至关重要的蛋白质的基因,由于大片段缺失而丢失。对患者成纤维细胞的功能分析表明,相应的等位基因携带 DCLRE1C 基因的无义突变。

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