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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Pubblicato in:Mol Ther Nucleic Acids
Autori principali: Dooley, Scott J., McDougald, Devin S., Fisher, Krishna J., Bennicelli, Jeanette L., Mitchell, Lloyd G., Bennett, Jean
Natura: Artigo
Lingua:Inglês
Pubblicazione: American Society of Gene & Cell Therapy 2018
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Article
Accesso online: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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