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RNA Editing as a Therapeutic Approach for Retinal Gene Therapy Requiring Long Coding Sequences

RNA editing aims to treat genetic disease through altering gene expression at the transcript level. Pairing site-directed RNA-targeting mechanisms with engineered deaminase enzymes allows for the programmable correction of G>A and T>C mutations in RNA. This offers a promising therapeutic appro...

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Detalhes bibliográficos
Publicado no:Int J Mol Sci
Main Authors: Fry, Lewis E., Peddle, Caroline F., Barnard, Alun R., McClements, Michelle E., MacLaren, Robert E.
Formato: Artigo
Idioma:Inglês
Publicado em: MDPI 2020
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC7037314/
https://ncbi.nlm.nih.gov/pubmed/31991730
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3390/ijms21030777
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