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Metabolic rescue in pluripotent cells from patients with mtDNA disease

Author

Listed:
  • Hong Ma

    (Center for Embryonic Cell and Gene Therapy, Oregon Health & Science University
    Oregon National Primate Research Center, 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
    Oregon National Primate Research Center, Oregon Health & Science University)

  • Eunju Kang

    (Center for Embryonic Cell and Gene Therapy, Oregon Health & Science University
    Oregon National Primate Research Center, Oregon Health & Science University)

  • Yeonmi Lee

    (Center for Embryonic Cell and Gene Therapy, Oregon Health & Science University
    Oregon National Primate Research Center, Oregon Health & Science University)

  • Tomonari Hayama

    (Center for Embryonic Cell and Gene Therapy, Oregon Health & Science University
    Oregon National Primate Research Center, Oregon Health & Science University)

  • Ying Li

    (Center for Embryonic Cell and Gene Therapy, Oregon Health & Science University
    Oregon National Primate Research Center, Oregon Health & Science University)

  • Crystal Van Dyken

    (Center for Embryonic Cell and Gene Therapy, Oregon Health & Science University
    Oregon National Primate Research Center, Oregon Health & Science University)

  • Nuria Marti Gutierrez

    (Center for Embryonic Cell and Gene Therapy, Oregon Health & Science University
    Oregon National Primate Research Center, Oregon Health & Science University)

  • Rebecca Tippner-Hedges

    (Center for Embryonic Cell and Gene Therapy, Oregon Health & Science University
    Oregon National Primate Research Center, Oregon Health & Science University)

  • Amy Koski

    (Center for Embryonic Cell and Gene Therapy, Oregon Health & Science University
    Oregon National Primate Research Center, Oregon Health & Science University)

  • Nargiz Mitalipov

    (Center for Embryonic Cell and Gene Therapy, Oregon Health & Science University
    Oregon National Primate Research Center, 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
    Oregon National Primate Research Center, Oregon Health & Science University)

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.

Suggested Citation

  • 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 & Y, 2015. "Metabolic rescue in pluripotent cells from patients with mtDNA disease," Nature, Nature, vol. 524(7564), pages 234-238, August.
    • Handle: RePEc:nat:nature:v:524:y:2015:i:7564:d:10.1038_nature14546
    DOI: 10.1038/nature14546
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