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Using a barcoded AAV capsid library to select for clinically relevant gene therapy vectors

13 Nov 2019

Abstract excerpt

While gene transfer using recombinant adeno-associated viral (rAAV) vectors has shown success in some clinical trials, there remain many tissues that are not well transduced. Because of the recent success in reprogramming islet-derived cells into functional β cells in animal models, we constructed 2 highly complex barcoded replication competent capsid shuffled libraries and selected for high-transducing variants...

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PMID: 31723052
DOI: 10.1172/jci.insight.131610

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