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...

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Detalles Bibliográficos
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
Formato: Online Artículo Texto
Lenguaje:English
Publicado: 2018
Materias:
Article
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

Internet

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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