Metabolic rescue in pluripotent cells from patients with mtDNA disease

Hong Ma, Clifford D. L. Folmes, Jun Wu, Robert Morey, Sergio Mora-Castilla, Alejandro Ocampo, Li Ma, Joanna Poulton, Xinjian Wang, Riffat Ahmed, Eunju Kang, Yeonmi Lee, Tomonari Hayama, Ying Li, Crystal Van Dyken, Nuria Marti Gutierrez, Rebecca Tippner-Hedges, Amy Koski, Nargiz Mitalipov, Paula Amato, Don P. Wolf, Taosheng Huang, Andre Terzic, Louise C. Laurent, Juan Carlos Izpisua Belmonte and Shoukhrat Mitalipov ()
Additional contact information
Hong Ma: Center for Embryonic Cell and Gene Therapy, Oregon Health & Science University
Clifford D. L. Folmes: Center for Regenerative Medicine and Department of Medicine, Division of Cardiovascular Diseases
Jun Wu: Gene Expression Laboratory, Salk Institute for Biological Studies
Robert Morey: University of California
Sergio Mora-Castilla: University of California
Alejandro Ocampo: Gene Expression Laboratory, Salk Institute for Biological Studies
Li Ma: Gene Expression Laboratory, Salk Institute for Biological Studies
Joanna Poulton: John Radcliffe Hospital, University of Oxford
Xinjian Wang: Cincinnati Children's Hospital Medical Center
Riffat Ahmed: Center for Embryonic Cell and Gene Therapy, Oregon Health & Science University
Eunju Kang: Center for Embryonic Cell and Gene Therapy, Oregon Health & Science University
Yeonmi Lee: Center for Embryonic Cell and Gene Therapy, Oregon Health & Science University
Tomonari Hayama: Center for Embryonic Cell and Gene Therapy, Oregon Health & Science University
Ying Li: Center for Embryonic Cell and Gene Therapy, Oregon Health & Science University
Crystal Van Dyken: Center for Embryonic Cell and Gene Therapy, Oregon Health & Science University
Nuria Marti Gutierrez: Center for Embryonic Cell and Gene Therapy, Oregon Health & Science University
Rebecca Tippner-Hedges: Center for Embryonic Cell and Gene Therapy, Oregon Health & Science University
Amy Koski: Center for Embryonic Cell and Gene Therapy, Oregon Health & Science University
Nargiz Mitalipov: Center for Embryonic Cell and Gene Therapy, Oregon Health & Science University
Paula Amato: Oregon Health and Science University
Don P. Wolf: Oregon National Primate Research Center, Oregon Health & Science University
Taosheng Huang: Cincinnati Children's Hospital Medical Center
Andre Terzic: Center for Regenerative Medicine and Department of Medicine, Division of Cardiovascular Diseases
Louise C. Laurent: University of California
Juan Carlos Izpisua Belmonte: Gene Expression Laboratory, Salk Institute for Biological Studies
Shoukhrat Mitalipov: Center for Embryonic Cell and Gene Therapy, Oregon Health & Science University

Nature, 2015, vol. 524, issue 7564, 234-238

Abstract: 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.

Date: 2015
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DOI: 10.1038/nature14546

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