A magic bullet to specifically eliminate mutated mitochondrial genomes from patients' cells

Abstract

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). In this issue of EMBO Molecular Medicine, mitochondrial-targeted zinc finger nucleases (mtZFN) are used to alter the mitochondrial DNA (mtDNA) heteroplasmy balance in cells derived from patients with mitochondrial diseases. © 2014 The Author.