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

Sandra R. Bacman, Sion Llewelyn Williams, Milena Pinto, Susana Peralta, Carlos T Moraes

Research output: Contribution to journalArticle

167 Citations (Scopus)

Abstract

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
Pages (from-to)1111-1113
Number of pages3
JournalNature Medicine
Volume19
Issue number9
DOIs
StatePublished - Sep 1 2013

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Mitochondrial Genome
Mitochondrial DNA
Genes
Mitochondrial Diseases
Mitochondria
Transcription
Mutation

ASJC Scopus subject areas

  • Biochemistry, Genetics and Molecular Biology(all)
  • Medicine(all)

Cite this

Specific elimination of mutant mitochondrial genomes in patient-derived cells by mitoTALENs. / Bacman, Sandra R.; Williams, Sion Llewelyn; Pinto, Milena; Peralta, Susana; Moraes, Carlos T.

In: Nature Medicine, Vol. 19, No. 9, 01.09.2013, p. 1111-1113.

Research output: Contribution to journalArticle

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