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...

Descripció completa

Guardat en:
Dades bibliogràfiques
Publicat a:Mol Ther Nucleic Acids
Autors principals: Dooley, Scott J., McDougald, Devin S., Fisher, Krishna J., Bennicelli, Jeanette L., Mitchell, Lloyd G., Bennett, Jean
Format: Artigo
Idioma:Inglês
Publicat: American Society of Gene & Cell Therapy 2018
Matèries:
Article
Accés en línia: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
Etiquetes: Afegir etiqueta
Sense etiquetes, Sigues el primer a etiquetar aquest registre!

Ítems similars