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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...
Autores principales: | Sinnamon, John R., Kim, Susan Y., Fisk, Jenna R., Song, Zhen, Nakai, Hiroyuki, Jeng, Sophia, McWeeney, Shannon K., Mandel, Gail |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
2020
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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 |
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