Department of Neurology and Neurosurgery, Universidade Federal de São Paulo, Escola Paulista de Medicina, São Paulo, Brazil.
J Neurol Sci. 2012 Sep 15;320(1-2):131-5. doi: 10.1016/j.jns.2012.05.037. Epub 2012 Jun 27.
We identified a double mutation in a patient with chronic progressive external ophthalmoplegia, located in the tRNA(Ala) (m.5628T>C) and tRNA(Lys) (m.8348A>G) genes. Both mutations were previously described separately and considered pathogenic, however the same mutations were also reported as polymorphisms or phenotype modulator. We analyzed the proportion of each mutation in isolated muscle fibers by single fiber-polymerase chain reaction to investigate the contribution of each mutation to mitochondrial deficiency. Our findings demonstrated that the mutations were heteroplasmic in skeletal muscle and both mutations were present in all single muscle fibers. The proportions of the m.5628T>C mutation were not significantly different between normal and cytochrome-c-oxidase (COX) deficient fibers. However, a significant higher proportion of the m.8348A>G mutation was observed in COX deficient fibers. Homoplasmic m.8348A>G was only observed in COX negative fibers. In conclusion, we provide a piece of evidence toward the pathogenicity of the m.8348A>G mutation and suggest that m.5628T>C is probably a neutral polymorphism.
我们在一位患有慢性进行性眼外肌麻痹的患者中发现了位于 tRNA(Ala) (m.5628T>C) 和 tRNA(Lys) (m.8348A>G) 基因中的双突变。这两种突变先前都分别被描述为致病性突变,但是相同的突变也被报道为多态性或表型调节剂。我们通过单纤维聚合酶链反应分析了孤立肌肉纤维中每种突变的比例,以研究每种突变对线粒体缺陷的贡献。我们的研究结果表明,突变在骨骼肌中为异质性,并且两种突变均存在于所有单个肌肉纤维中。m.5628T>C 突变的比例在正常和细胞色素 c 氧化酶 (COX) 缺陷纤维之间没有显著差异。然而,在 COX 缺陷纤维中观察到 m.8348A>G 突变的比例显著更高。同质 m.8348A>G 仅在 COX 阴性纤维中观察到。总之,我们提供了支持 m.8348A>G 突变致病性的证据,并表明 m.5628T>C 可能是一种中性多态性。