Effective delivery of large genes to the retina by dual AAV vectors

Retinal gene therapy with adeno-associated viral (AAV) vectors is safe and effective in humans. However, AAV's limited cargo capacity prevents its application to therapies of inherited retinal diseases due to mutations of genes over 5 kb, like Stargardt's disease (STGD) and Usher syndrome...

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Bibliographische Detailangaben
Hauptverfasser: Trapani, Ivana, Colella, Pasqualina, Sommella, Andrea, Iodice, Carolina, Cesi, Giulia, de Simone, Sonia, Marrocco, Elena, Rossi, Settimio, Giunti, Massimo, Palfi, Arpad, Farrar, Gwyneth J, Polishchuk, Roman, Auricchio, Alberto
Format: Artigo
Sprache:Inglês
Veröffentlicht: Blackwell Publishing Ltd 2014
Schlagworte:
Research Article
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC3927955/
https://ncbi.nlm.nih.gov/pubmed/24150896
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/emmm.201302948
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