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Metabolic rescue in pluripotent cells from patients with mtDNA disease
- Ma, Hong;
- Folmes, Clifford DL;
- Wu, Jun;
- Morey, Robert;
- Mora-Castilla, Sergio;
- Ocampo, Alejandro;
- Ma, Li;
- Poulton, Joanna;
- Wang, Xinjian;
- Ahmed, Riffat;
- Kang, Eunju;
- Lee, Yeonmi;
- Hayama, Tomonari;
- Li, Ying;
- Van Dyken, Crystal;
- Gutierrez, Nuria Marti;
- Tippner-Hedges, Rebecca;
- Koski, Amy;
- Mitalipov, Nargiz;
- Amato, Paula;
- Wolf, Don P;
- Huang, Taosheng;
- Terzic, Andre;
- Laurent, Louise C;
- Belmonte, Juan Carlos Izpisua;
- Mitalipov, Shoukhrat
Published Web Location
https://doi.org/10.1038/nature14546Abstract
Mutations in mitochondrial (mt)DNA are associated with severe disorders for which treatment is currently limited; this study shows that mtDNA mutations can be genetically corrected and normal metabolic function restored in cells derived from patients with mtDNA disease and reprogrammed to pluripotency through factor-mediated reprogramming or via a somatic cell nuclear transfer approach.
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