Department of Biological Chemistry, John Innes Centre, Norwich NR4 7UH, UK.
School of Biological Sciences, University of East Anglia, Norwich NR4 7TJ, UK.
Hum Mol Genet. 2018 Nov 1;27(21):3697-3709. doi: 10.1093/hmg/ddy247.
Complex I deficiency is a common cause of mitochondrial disease, resulting from mutations in genes encoding structural subunits, assembly factors or defects in mitochondrial gene expression. Advances in genetic diagnostics and sequencing have led to identification of several variants in NUBPL (nucleotide binding protein-like), encoding an assembly factor of complex I, which are potentially pathogenic. To help assign pathogenicity and learn more about the function of NUBPL, amino acid substitutions were recreated in the homologous Ind1 protein of the yeast model Yarrowia lipolytica. Leu102Pro destabilized the Ind1 protein, leading to a null-mutant phenotype. Asp103Tyr, Leu191Phe and Gly285Cys affected complex I assembly to varying degrees, whereas Gly136Asp substitution in Ind1 did not impact on complex I levels nor dNADH:ubiquinone activity. Blue-native polyacrylamide gel electrophoresis and immunolabelling of the structural subunits NUBM and NUCM revealed that all Ind1 variants accumulated a Q module intermediate of complex I. In the Ind1 Asp103Tyr variant, the matrix arm intermediate was virtually absent, indicating a dominant effect. Dysfunction of Ind1, but not absence of complex I, rendered Y. lipolytica sensitive to cold. The Ind1 Gly285Cys variant was able to support complex I assembly at 28°C, but not at 10°C. Our results indicate that Ind1 is required for progression of assembly from the Q module to the full matrix arm. Cold sensitivity could be developed as a phenotype assay to demonstrate pathogenicity of NUBPL mutations and other complex I defects.
复合体 I 缺陷是一种常见的线粒体疾病病因,由编码结构亚基、组装因子的基因突变或线粒体基因表达缺陷引起。遗传诊断和测序技术的进步已经鉴定出编码复合体 I 组装因子的 NUBPL(核苷酸结合蛋白样)基因中的几个变体,这些变体可能具有致病性。为了帮助确定致病性并更多地了解 NUBPL 的功能,在酵母模型酿酒酵母的同源 Ind1 蛋白中重新创建了氨基酸取代。Leu102Pro 使 Ind1 蛋白不稳定,导致缺失突变表型。Asp103Tyr、Leu191Phe 和 Gly285Cys 对复合体 I 组装的影响程度不同,而 Ind1 中的 Gly136Asp 取代对复合体 I 水平和 dNADH:泛醌活性没有影响。蓝色非变性聚丙烯酰胺凝胶电泳和结构亚基 NUBM 和 NUCM 的免疫标记显示,所有 Ind1 变体都积累了复合体 I 的 Q 模块中间产物。在 Ind1 Asp103Tyr 变体中,基质臂中间产物几乎不存在,表明存在显性效应。Ind1 的功能障碍而不是复合体 I 的缺失使酿酒酵母对冷敏感。Ind1 Gly285Cys 变体能够在 28°C 下支持复合体 I 组装,但不能在 10°C 下支持。我们的结果表明,Ind1 是从 Q 模块到完整基质臂组装的进展所必需的。冷敏感性可以作为表型测定开发,以证明 NUBPL 突变和其他复合体 I 缺陷的致病性。