Ahola-Erkkilä · Human molecular genetics 2010 · controlled animal experiment · n=?

Ketogenic diet slows down mitochondrial myopathy progression in mice.

Cited 192 times in the scientific literature.

Level 5 - mechanism / opinion, no new human data

Animal model study (transgenic mice)

PubMed 20167576 · doi:10.1093/hmg/ddq076 · record verified 2026-08-30

What was done

Transgenic Deletor mice, an animal model of progressive late-onset mitochondrial myopathy driven by mitochondrial DNA (mtDNA) deletions and respiratory chain deficiency, were treated with a ketogenic diet (low glucose, high fat) administered either as a long-term pre-symptomatic regimen or a shorter-term post-symptomatic regimen. Effects on disease progression were evaluated using morphological, metabolomic, and lipidomic profiling.

What was found

The abstract reports qualitative outcomes without specific numerical values. Ketogenic diet treatment decreased the frequency of cytochrome c oxidase-negative muscle fibers and completely prevented mitochondrial ultrastructural abnormalities in skeletal muscle. The diet restored most altered plasma phospholipids, free amino acids, and metabolomic/lipidomic markers to wild-type levels, normalized liver lipid levels, and induced mitochondrial biogenesis. It did not significantly alter mtDNA quality or quantity.

Why it matters

These findings suggest that dietary modulation via a ketogenic diet can slow histological and metabolic progression in mitochondrial myopathy by promoting mitochondrial biogenesis rather than clearing mutant mtDNA, offering a potential therapeutic strategy for human late-onset mitochondrial disorders.

Limits

Findings are restricted to a transgenic mouse model and cannot be directly translated to human clinical efficacy or safety. The abstract does not provide quantitative data, exact sample sizes, dietary compositions, treatment durations, or functional/behavioral outcome metrics.

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