Specific elimination of mutant mitochondrial genomes in patient-derived cells by mitoTALENs

Sandra R. Bacman, Siôn L. Williams, Milena Pinto, Susana Peralta, Carlos T. Moraes

Research output: Contribution to journalArticle

187 Scopus citations


Mitochondrial diseases are commonly caused by mutated mitochondrial DNA (mtDNA), which in most cases coexists with wild-type mtDNA, resulting in mtDNA heteroplasmy. We have engineered transcription activator-like effector nucleases (TALENs) to localize to mitochondria and cleave different classes of pathogenic mtDNA mutations. Mitochondria-targeted TALEN (mitoTALEN) expression led to permanent reductions in deletion or point-mutant mtDNA in patient-derived cells, raising the possibility that these mitochondrial nucleases can be therapeutic for some mitochondrial diseases.

Original languageEnglish (US)
Pages (from-to)1111-1113
Number of pages3
JournalNature medicine
Issue number9
StatePublished - Sep 1 2013


ASJC Scopus subject areas

  • Biochemistry, Genetics and Molecular Biology(all)

Cite this