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In Vivo Repair of a Protein Underlying a Neurological Disorder by Programmable RNA Editing

Programmable RNA editing is gaining momentum as an approach to repair mutations, but its efficiency in repairing endogenous mutant RNA in complex tissue is unknown. Here we apply this approach to the brain and successfully repair a guanosine-to-adenosine mutation in methyl CpG binding protein 2 RNA...

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Detalles Bibliográficos
Autores principales: Sinnamon, John R., Kim, Susan Y., Fisk, Jenna R., Song, Zhen, Nakai, Hiroyuki, Jeng, Sophia, McWeeney, Shannon K., Mandel, Gail
Formato: Online Artículo Texto
Lenguaje:English
Publicado: 2020
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7449137/
https://www.ncbi.nlm.nih.gov/pubmed/32668243
http://dx.doi.org/10.1016/j.celrep.2020.107878