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Transcription activator-like effector nucleases (TALENs) to eliminate mitochondria-specific mutant DNA

TALENs could be useful for treating mitochondrial disease by selectively removing mutant mitochondrial DNA (mtDNA). In cells with a pathogenic point-of-deletion mutation in mtDNA, transient transfection of plasmids expressing mitochondria-targeted TALENs decreased levels of the mutant but not wild-type mtDNA. Next steps include developing efficient TALEN delivery strategies and testing the method in mouse models of mitochondrial disease.

SciBX 6(34); doi:10.1038/scibx.2013.944
Published online Sept. 5, 2013

Patenting status undisclosed; licensing details available from the University of Miami

Bacman, S.R. et al. Nat. Med.; published online Aug. 4, 2013;
Contact: Carlos T. Moraes,
University of Miami, Miami, Fla.