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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Vydáno v:Mol Ther Nucleic Acids
Hlavní autoři: Dooley, Scott J., McDougald, Devin S., Fisher, Krishna J., Bennicelli, Jeanette L., Mitchell, Lloyd G., Bennett, Jean
Médium: Artigo
Jazyk:Inglês
Vydáno: American Society of Gene & Cell Therapy 2018
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Article
On-line přístup: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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