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Article

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

9 Apr 2020

Abstract excerpt

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 library - to create AAV viral capsids with the capability to deliver genes to the outer retina in primates. A...

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PMID: 32271719
DOI: 10.1172/jci.insight.135112

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