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Genome editing in mitochondria corrects a pathogenic mtDNA mutation in vivo
Mutations of the mitochondrial genome (mtDNA) underlie a significant portion of mitochondrial disease burden. These disorders are currently incurable and effectively untreatable, with heterogeneous penetrance, presentation and prognosis. To address the lack of effective treatment for these disorders...
Autores principales: | Gammage, Payam A., Viscomi, Carlo, Simard, Marie-Lune, Costa, Ana S.H., Gaude, Edoardo, Powell, Christopher A., Van Haute, Lindsey, McCann, Beverly J., Rebelo-Guiomar, Pedro, Cerutti, Raffaele, Zhang, Lei, Rebar, Edward J., Zeviani, Massimo, Frezza, Christian, Stewart, James B., Minczuk, Michal |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
2018
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6225988/ https://www.ncbi.nlm.nih.gov/pubmed/30250142 http://dx.doi.org/10.1038/s41591-018-0165-9 |
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