See also: PA Gammage et al (April 2014)
A magic bullet to specifically eliminate mutated mitochondrial genomes from patients' cells
Article first published online: 12 MAR 2014
© 2014 The Author. Published under the terms of the CC BY license.
This is an open access article under the terms of the Creative Commons Attribution License, which permits use, distribution and reproduction in any medium, provided the original work is properly cited.
EMBO Molecular Medicine
Volume 6, Issue 4, pages 434–435, April 2014
How to Cite
Moraes, C. T. (2014), A magic bullet to specifically eliminate mutated mitochondrial genomes from patients' cells. EMBO Mol Med, 6: 434–435. doi: 10.1002/emmm.201303769
- Issue published online: 2 APR 2014
- Article first published online: 12 MAR 2014
When mitochondrial diseases result from mutations found in the mitochondrial DNA, engineered mitochondrial-targeted nucleases such as mitochondrial-targeted zinc finger nucleases are shown to specifically eliminate the mutated molecules, leaving the wild-type mitochondrial DNA intact to replicate and restore normal copy number. In this issue, Gammage and colleagues successfully apply this improved technology on patients' cells with two types of genetic alterations responsible for neuropathy ataxia and retinitis pigmentosa (NARP) syndrome and Kearns Sayre syndrome and progressive external ophthalmoplegia (PEO).