Shepherd R K, Checcarelli N, Naini A, De Vivo D C, DiMauro S, Sue C M
Department of Neurogenetics, Kolling Institute of Medical Research, Royal North Shore Hospital and University of Sydney, St Leonard's, NSW, Australia.
J Inherit Metab Dis. 2006 Feb;29(1):86-91. doi: 10.1007/s10545-006-0148-8.
Mitochondrial diseases are a heterogeneous group of disorders caused by mutations in both nuclear DNA (nDNA) and mitochondrial DNA (mtDNA). Mitochondrial disease leads to impaired respiratory chain function and reduced ATP production. The aim of this study was to compare disturbances in mitochondrial function by measuring ATP synthesis in fibroblasts derived from patients with nDNA and mtDNA defects. Skin fibroblasts derived from 22 patients with either nDNA-related disorders (n = 8) or mtDNA-related disorders (n = 14) were analysed. ATP synthesis was markedly decreased in fibroblasts derived from patients with nDNA-related disorders but only variably so in patients with mtDNA mutations. In fibroblasts with the MELAS 3243A > G mutation, ATP synthesis correlated with mutant load. We believe that the observed differences in ATP production between cell lines derived from patients with nDNA-related disorders and mtDNA-related disorders may help in the assessment of patients with undiagnosed mitochondrial disease. The clinical comparisons observed in patients with nDNA- and mtDNA-related disorders may be explained by differences in the disturbance of ATP synthesis measured in the two conditions.
线粒体疾病是由核DNA(nDNA)和线粒体DNA(mtDNA)突变引起的一组异质性疾病。线粒体疾病导致呼吸链功能受损和ATP生成减少。本研究的目的是通过测量来自nDNA和mtDNA缺陷患者的成纤维细胞中的ATP合成,比较线粒体功能的紊乱情况。对来自22例患有nDNA相关疾病(n = 8)或mtDNA相关疾病(n = 14)患者的皮肤成纤维细胞进行了分析。来自nDNA相关疾病患者的成纤维细胞中ATP合成明显减少,但在mtDNA突变患者中仅有所不同。在具有MELAS 3243A>G突变的成纤维细胞中,ATP合成与突变负荷相关。我们认为,在来自nDNA相关疾病患者和mtDNA相关疾病患者的细胞系中观察到的ATP生成差异可能有助于评估未确诊的线粒体疾病患者。在nDNA和mtDNA相关疾病患者中观察到的临床差异可能由两种情况下测量的ATP合成紊乱差异来解释。