Spliceosome-Mediated Pre-mRNA trans-Splicing Can Repair CEP290 mRNA

Ocular gene therapy with recombinant adeno-associated virus (AAV) has shown vector-mediated gene augmentation to be safe and efficacious in the retina in one set of diseases (retinitis pigmentosa and Leber congenital amaurosis (LCA) caused by RPE65 deficiency), with excellent safety profiles to date...

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Publicado en:Mol Ther Nucleic Acids
Main Authors: Dooley, Scott J., McDougald, Devin S., Fisher, Krishna J., Bennicelli, Jeanette L., Mitchell, Lloyd G., Bennett, Jean
Formato: Artigo
Idioma:Inglês
Publicado: American Society of Gene & Cell Therapy 2018
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Article
Acceso en liña:https://ncbi.nlm.nih.gov/pmc/articles/PMC6023944/
https://ncbi.nlm.nih.gov/pubmed/30195768
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtn.2018.05.014
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