In vivo–directed evolution of adeno-associated virus in the primate retina

Efficient adeno-associated virus–mediated (AAV-mediated) gene delivery remains a significant obstacle to effective retinal gene therapies. Here, we apply directed evolution — guided by deep sequencing and followed by direct in vivo secondary selection of high-performing vectors with a GFP-barcoded l...

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Veröffentlicht in:JCI Insight
Hauptverfasser: Byrne, Leah C., Day, Timothy P., Visel, Meike, Strazzeri, Jennifer A., Fortuny, Cécile, Dalkara, Deniz, Merigan, William H., Schaffer, David V., Flannery, John G.
Format: Artigo
Sprache:Inglês
Veröffentlicht: American Society for Clinical Investigation 2020
Schlagworte:
Research Article
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC7259523/
https://ncbi.nlm.nih.gov/pubmed/32271719
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1172/jci.insight.135112
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