Article
MitoTALEN: A General Approach to Reduce Mutant mtDNA Loads and Restore Oxidative Phosphorylation Function in Mitochondrial Diseases.
Molecular therapy : the journal of the American Society of Gene Therapy - 1 Oct 2015
Hashimoto Masami, Bacman Sandra R, Peralta Susana, Falk Marni J, Chomyn Anne, Chan David C, Williams Sion L, Moraes Carlos T
Abstract excerpt
We have designed mitochondrially targeted transcription activator-like effector nucleases or mitoTALENs to cleave specific sequences in the mitochondrial DNA (mtDNA) with the goal of eliminating mtDNA carrying pathogenic point mutations. To test the generality of the approach, we designed mitoTALENs to target two relatively common pathogenic mtDNA point mutations associated with mitochondrial diseases: the...
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