MitoTALEN: A General Approach to Reduce Mutant mtDNA Loads and Restore Oxidative Phosphorylation Function in Mitochondrial Diseases

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...

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Vydáno v:Mol Ther
Hlavní autoři: Hashimoto, Masami, Bacman, Sandra R, Peralta, Susana, Falk, Marni J, Chomyn, Anne, Chan, David C, Williams, Sion L, Moraes, Carlos T
Médium: Artigo
Jazyk:Inglês
Vydáno: Nature Publishing Group 2015
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Original Article
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC4817924/
https://ncbi.nlm.nih.gov/pubmed/26159306
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2015.126
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