Malycha Friederike, Eggermann Thomas, Hristov Mihail, Schena Francesco Paolo, Mertens Peter R, Zerres Klaus, Floege Jürgen, Eitner Frank
Department of Human Genetics, Division of Nephrology and Immunology, Aachen, Germany.
Nephrol Dial Transplant. 2009 Jan;24(1):321-4. doi: 10.1093/ndt/gfn538. Epub 2008 Oct 7.
Altered IgA1 galactosylation is involved in the pathogenesis of IgA nephropathy (IgAN). The galactosyltransferase core-1 beta3-galactosyltransferase-1 (C1GALT1) and its chaperone cosmc are specifically required for O-galactosylation of the IgA1 hinge region. Mutations in the cosmc gene result in a secondary loss of function of C1GALT1 with subsequent undergalactosylation of glycoproteins. Mosaic mutations of cosmc have been shown to result in autoimmune disease. We hypothesized that cosmc mutations might contribute to the altered IgA1 galactosylation in IgAN patients.
We studied cosmc gene sequences in genomic DNA obtained from male patients with biopsy-proven sporadic (n = 33) and familial IgAN (n = 6 patients from different families). To account for a potential mosaicism we sequenced cosmc in 10 different peripheral blood mononuclear cell DNA clones of every patient. To specifically assess potential mosaic mutations in IgA-producing cells, cosmc mutations were also analysed in DNA isolated from CD20+ B-lymphocytes from three male IgAN patients.
Despite our extensive genomic analysis, the data revealed no functionally relevant cosmc gene variants in sporadic or familial IgAN cases. A cosmc gene polymorphism, rs17261572, was identified in these IgAN patients in a similar frequency as previously reported in healthy adults. A functional consequence of this polymorphism has not yet been determined.
Although decreased C1GALT1 activity has been implicated in the IgAN pathogenesis and cosmc chaperone mutations can cause autoimmune disease, our data provide no evidence for a relevant role of cosmc gene mutations in European patients with sporadic or familial IgAN.
IgA1半乳糖基化改变参与IgA肾病(IgAN)的发病机制。IgA1铰链区O-半乳糖基化特别需要半乳糖基转移酶核心1β3-半乳糖基转移酶-1(C1GALT1)及其伴侣分子cosmc。cosmc基因突变导致C1GALT1继发性功能丧失,随后糖蛋白半乳糖基化不足。已表明cosmc的镶嵌突变会导致自身免疫性疾病。我们推测cosmc突变可能导致IgAN患者IgA1半乳糖基化改变。
我们研究了经活检证实的散发性(n = 33)和家族性IgAN(来自不同家族的6例患者)男性患者基因组DNA中的cosmc基因序列。为了考虑潜在的镶嵌现象,我们对每位患者的10个不同外周血单个核细胞DNA克隆中的cosmc进行了测序。为了特异性评估产生IgA细胞中的潜在镶嵌突变,还分析了3例男性IgAN患者CD20+B淋巴细胞分离的DNA中的cosmc突变。
尽管我们进行了广泛的基因组分析,但数据显示散发性或家族性IgAN病例中没有功能相关的cosmc基因变异。在这些IgAN患者中鉴定出一种cosmc基因多态性rs17261572,其频率与先前在健康成年人中报道的相似。尚未确定这种多态性的功能后果。
尽管C1GALT1活性降低与IgAN发病机制有关,且cosmc伴侣分子突变可导致自身免疫性疾病,但我们的数据没有提供证据表明cosmc基因突变在欧洲散发性或家族性IgAN患者中起相关作用。