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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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Bibliografske podrobnosti
izdano v:Mol Ther
Main Authors: Hashimoto, Masami, Bacman, Sandra R, Peralta, Susana, Falk, Marni J, Chomyn, Anne, Chan, David C, Williams, Sion L, Moraes, Carlos T
Format: Artigo
Jezik:Inglês
Izdano: Nature Publishing Group 2015
Teme:
Online dostop: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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