title: Specific elimination of mutant mitochondrial genomes in patient-derived cells by mitoTALENs.
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.
authors.affiliation: 1] Department of Neurology, University of Miami Miller School of Medicine, Miami, Florida, USA. [2].
authors.forename: Sandra R
authors.initials: SR
authors.lastname: Bacman
authors.forename: Siôn L
authors.initials: SL
authors.lastname: Williams
authors.forename: Milena
authors.initials: M
authors.lastname: Pinto
authors.forename: Susana
authors.initials: S
authors.lastname: Peralta
authors.forename: Carlos T
authors.initials: CT
authors.lastname: Moraes
mesh_terms: D000595:Amino Acid Sequence; D045744:Cell Line, Tumor; D004272:DNA, Mitochondrial / Q000235:genetics*; D015252:Deoxyribonucleases, Type II Site-Specific / Q000378:metabolism* / Q000494:pharmacology; D054629:Genome, Mitochondrial; D006801:Humans; D008928:Mitochondria / Q000235:genetics; D028361:Mitochondrial Diseases / Q000188:drug therapy / Q000235:genetics*; D008969:Molecular Sequence Data; D009154:Mutation; D012516:Osteosarcoma / Q000188:drug therapy / Q000235:genetics*
chemical_list: D004272:DNA, Mitochondrial; D015252:Deoxyribonucleases, Type II Site-Specific
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