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J Inherit Metab Dis. 2011 Apr;34(2):309-13. doi: 10.1007/s10545-010-9122-6. Epub 2010 May 27.

Altering the balance between healthy and mutated mitochondrial DNA.

Author information

1
Mitochondrial Research Group, Institute of Ageing and Health, Medical School, Framlington Place, Newcastle upon Tyne, UK. Paul.smith@ncl.ac.uk

Abstract

Pathogenic mutations of the mitochondrial genome are frequently found to co-exist with wild-type mtDNA molecules, a state known as heteroplasmy. In most disease cases, the mutation is recessive with manifestation of a clinical phenotype occurring when the proportion of mutated mtDNA exceeds a high threshold. The concept of increasing the ratio of healthy to mutated mtDNA as a means to correcting the biochemical defect has received much attention. A number of strategies are highlighted in this article, including manipulation of the mitochondrial genome by antigenomic drugs or restriction endonucleases, zinc finger peptide-targeted nucleases and exercise-induced gene shifting. The feasibility of these approaches has been demonstrated in a number of models, however more work is necessary before use in human patients.

PMID:
20506041
DOI:
10.1007/s10545-010-9122-6
[Indexed for MEDLINE]
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