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补体因子H相关基因CFHR1和CFHR3的缺失与非典型溶血尿毒综合征相关。

Deletion of complement factor H-related genes CFHR1 and CFHR3 is associated with atypical hemolytic uremic syndrome.

作者信息

Zipfel Peter F, Edey Matthew, Heinen Stefan, Józsi Mihály, Richter Heiko, Misselwitz Joachim, Hoppe Bernd, Routledge Danny, Strain Lisa, Hughes Anne E, Goodship Judith A, Licht Christoph, Goodship Timothy H J, Skerka Christine

机构信息

Leibniz Institute for Natural Product Research and Infection Biology, Hans Knoell Institute, Jena, Germany.

出版信息

PLoS Genet. 2007 Mar 16;3(3):e41. doi: 10.1371/journal.pgen.0030041. Epub 2007 Feb 1.

Abstract

Atypical hemolytic uremic syndrome (aHUS) is associated with defective complement regulation. Disease-associated mutations have been described in the genes encoding the complement regulators complement factor H, membrane cofactor protein, factor B, and factor I. In this study, we show in two independent cohorts of aHUS patients that deletion of two closely related genes, complement factor H-related 1 (CFHR1) and complement factor H-related 3 (CFHR3), increases the risk of aHUS. Amplification analysis and sequencing of genomic DNA of three affected individuals revealed a chromosomal deletion of approximately 84 kb in the RCA gene cluster, resulting in loss of the genes coding for CFHR1 and CFHR3, but leaving the genomic structure of factor H intact. The CFHR1 and CFHR3 genes are flanked by long homologous repeats with long interspersed nuclear elements (retrotransposons) and we suggest that nonallelic homologous recombination between these repeats results in the loss of the two genes. Impaired protection of erythrocytes from complement activation is observed in the serum of aHUS patients deficient in CFHR1 and CFHR3, thus suggesting a regulatory role for CFHR1 and CFHR3 in complement activation. The identification of CFHR1/CFHR3 deficiency in aHUS patients may lead to the design of new diagnostic approaches, such as enhanced testing for these genes.

摘要

非典型溶血尿毒综合征(aHUS)与补体调节缺陷有关。在编码补体调节蛋白补体因子H、膜辅助蛋白、因子B和因子I的基因中已描述了与疾病相关的突变。在本研究中,我们在两个独立的aHUS患者队列中发现,两个密切相关的基因——补体因子H相关蛋白1(CFHR1)和补体因子H相关蛋白3(CFHR3)的缺失增加了aHUS的发病风险。对三名受影响个体的基因组DNA进行扩增分析和测序,发现RCA基因簇中存在约84 kb的染色体缺失,导致编码CFHR1和CFHR3的基因丢失,但因子H的基因组结构保持完整。CFHR1和CFHR3基因两侧是带有长散在核元件(反转录转座子)的长同源重复序列,我们认为这些重复序列之间的非等位基因同源重组导致了这两个基因的缺失。在缺乏CFHR1和CFHR3的aHUS患者血清中观察到红细胞免受补体激活的保护作用受损,因此提示CFHR1和CFHR3在补体激活中具有调节作用。在aHUS患者中鉴定出CFHR1/CFHR3缺陷可能会导致设计新的诊断方法,如加强对这些基因的检测。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/3c82/1839139/a5cc640a120c/pgen.0030041.g001.jpg

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