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Therapeutic Manipulation of mtDNA Heteroplasmy: A Shifting Perspective.
Trends Mol Med. 2020 Jul; 26(7):698-709.TM

Abstract

Mutations of mitochondrial DNA (mtDNA) often underlie mitochondrial disease, one of the most common inherited metabolic disorders. Since the sequencing of the human mitochondrial genome and the discovery of pathogenic mutations in mtDNA more than 30 years ago, a movement towards generating methods for robust manipulation of mtDNA has ensued, although with relatively few advances and some controversy. While developments in the transformation of mammalian mtDNA have stood still for some time, recent demonstrations of programmable nuclease-based technology suggest that clinical manipulation of mtDNA heteroplasmy may be on the horizon for these largely untreatable disorders. Here we review historical and recent developments in mitochondrially targeted nuclease technology and the clinical outlook for treatment of hereditary mitochondrial disease.

Authors+Show Affiliations

Stem Cells and Metabolism, Biomedicum Helsinki, University of Helsinki, Helsinki, Finland.Wellcome Centre for Mitochondrial Research, Translational and Clinical Research Institute, Newcastle University, Newcastle upon Tyne, UK.MRC Mitochondrial Biology Unit, School of Clinical Medicine, University of Cambridge, Cambridge, UK.CRUK Beatson Institute, Glasgow, UK; Institute of Cancer Sciences, University of Glasgow, Glasgow, UK. Electronic address: payam.gammage@glasgow.ac.uk.

Pub Type(s)

Journal Article
Review

Language

eng

PubMed ID

32589937

Citation

Jackson, Christopher B., et al. "Therapeutic Manipulation of mtDNA Heteroplasmy: a Shifting Perspective." Trends in Molecular Medicine, vol. 26, no. 7, 2020, pp. 698-709.
Jackson CB, Turnbull DM, Minczuk M, et al. Therapeutic Manipulation of mtDNA Heteroplasmy: A Shifting Perspective. Trends Mol Med. 2020;26(7):698-709.
Jackson, C. B., Turnbull, D. M., Minczuk, M., & Gammage, P. A. (2020). Therapeutic Manipulation of mtDNA Heteroplasmy: A Shifting Perspective. Trends in Molecular Medicine, 26(7), 698-709. https://doi.org/10.1016/j.molmed.2020.02.006
Jackson CB, et al. Therapeutic Manipulation of mtDNA Heteroplasmy: a Shifting Perspective. Trends Mol Med. 2020;26(7):698-709. PubMed PMID: 32589937.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Therapeutic Manipulation of mtDNA Heteroplasmy: A Shifting Perspective. AU - Jackson,Christopher B, AU - Turnbull,Doug M, AU - Minczuk,Michal, AU - Gammage,Payam A, Y1 - 2020/03/26/ PY - 2019/12/17/received PY - 2020/02/19/revised PY - 2020/02/21/accepted PY - 2020/6/27/entrez PY - 2020/6/27/pubmed PY - 2020/6/27/medline KW - gene therapy KW - heteroplasmy KW - mitoTALEN KW - mitochondrial disease KW - mtDNA KW - mtZFN SP - 698 EP - 709 JF - Trends in molecular medicine JO - Trends Mol Med VL - 26 IS - 7 N2 - Mutations of mitochondrial DNA (mtDNA) often underlie mitochondrial disease, one of the most common inherited metabolic disorders. Since the sequencing of the human mitochondrial genome and the discovery of pathogenic mutations in mtDNA more than 30 years ago, a movement towards generating methods for robust manipulation of mtDNA has ensued, although with relatively few advances and some controversy. While developments in the transformation of mammalian mtDNA have stood still for some time, recent demonstrations of programmable nuclease-based technology suggest that clinical manipulation of mtDNA heteroplasmy may be on the horizon for these largely untreatable disorders. Here we review historical and recent developments in mitochondrially targeted nuclease technology and the clinical outlook for treatment of hereditary mitochondrial disease. SN - 1471-499X UR - https://www.unboundmedicine.com/medline/citation/32589937/Therapeutic_Manipulation_of_mtDNA_Heteroplasmy:_A_Shifting_Perspective L2 - https://linkinghub.elsevier.com/retrieve/pii/S1471-4914(20)30062-9 DB - PRIME DP - Unbound Medicine ER -
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