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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Bibliographische Detailangaben
Veröffentlicht in:Mol Ther Nucleic Acids
Hauptverfasser: Dooley, Scott J., McDougald, Devin S., Fisher, Krishna J., Bennicelli, Jeanette L., Mitchell, Lloyd G., Bennett, Jean
Format: Artigo
Sprache:Inglês
Veröffentlicht: American Society of Gene & Cell Therapy 2018
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Article
Online Zugang: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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