Near-complete elimination of mutant mtDNA by iterative or dynamic dose-controlled treatment with mtZFNs

Mitochondrial diseases are frequently associated with mutations in mitochondrial DNA (mtDNA). In most cases, mutant and wild-type mtDNAs coexist, resulting in heteroplasmy. The selective elimination of mutant mtDNA, and consequent enrichment of wild-type mtDNA, can rescue pathological phenotypes in...

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Detalles Bibliográficos
Autores principales: Gammage, Payam A., Gaude, Edoardo, Van Haute, Lindsey, Rebelo-Guiomar, Pedro, Jackson, Christopher B., Rorbach, Joanna, Pekalski, Marcin L., Robinson, Alan J., Charpentier, Marine, Concordet, Jean-Paul, Frezza, Christian, Minczuk, Michal
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Oxford University Press 2016
Materias:
Molecular Biology
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5027515/
https://www.ncbi.nlm.nih.gov/pubmed/27466392
http://dx.doi.org/10.1093/nar/gkw676

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