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

EMBO Mol Med. 2014 Apr;6(4):434-5. doi: 10.1002/emmm.201303769. Epub 2014 Mar 12.


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).

Publication types

  • Comment

MeSH terms

  • DNA, Mitochondrial / metabolism*
  • Genetic Therapy*
  • Genome, Mitochondrial*
  • Humans
  • Mitochondria / enzymology*
  • Mitochondrial Diseases / genetics*
  • Mitochondrial Diseases / therapy*
  • Point Mutation*
  • Sequence Deletion*


  • DNA, Mitochondrial