A high mutation load of m.14597A>G in MT-ND6 causes Leigh syndrome

Sci Rep. 2021 May 27;11(1):11123. doi: 10.1038/s41598-021-90196-5.

Abstract

Leigh syndrome (LS) is an early-onset progressive neurodegenerative disorder associated with mitochondrial deficiency. m.14597A>G (p.Ile26Thr) in the MT-ND6 gene was reported to cause Leber's hereditary optic neuropathy (LHON) or dementia/dysarthria. In previous reports, less than 90% heteroplasmy was shown to result in adult-onset disease. Here, by whole mitochondrial sequencing, we identified m.14597A>G mutation of a patient with LS. PCR-RFLP analysis on fibroblasts from the patient revealed a high mutation load (> 90% heteroplasmy). We performed functional assays using cybrid cell models generated by fusing mtDNA-less rho0 HeLa cells with enucleated cells from patient fibroblasts carrying the m.14597A>G variant. Cybrid cell lines bearing the m.14597A>G variant exhibited severe effects on mitochondrial complex I activity. Additionally, impairment of cell proliferation, decreased ATP production and reduced oxygen consumption rate were observed in the cybrid cell lines bearing the m.14597A>G variant when the cells were metabolically stressed in medium containing galactose, indicating mitochondrial respiratory chain defects. These results suggest that a high mutation load of m.14597A>G leads to LS via a mitochondrial complex I defect, rather than LHON or dementia/dysarthria.

Publication types

  • Case Reports
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Electron Transport Complex I / genetics
  • Electron Transport Complex I / metabolism
  • Fibroblasts
  • Genes, Mitochondrial
  • HeLa Cells
  • Humans
  • Infant
  • Leigh Disease / genetics*
  • Leigh Disease / metabolism
  • Male
  • Mitochondria / genetics*
  • Mitochondria / metabolism
  • Mutation*
  • NADH Dehydrogenase / genetics*
  • NADH Dehydrogenase / metabolism
  • Oxygen Consumption / genetics

Substances

  • MT-ND6 protein, human
  • NADH Dehydrogenase
  • Electron Transport Complex I