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- W2955925866 abstract "ABSTRACT: While gene transfer using recombinant adeno-associated viral (rAAV) vectors have 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 two highly complex barcoded replication competent capsid shuffled libraries and selected for high transducing variants on primary human islets. We describe a chimeric capsid (AAV-KP1) that penetrated and transduced primary human islet cells and human embryonic stem cell derived β-cells with up to 10-fold higher efficiency compared to previously studied best in class AAV vectors. Remarkably, this chimeric capsid was also able to transduce both mouse and human hepatocytes at very high levels in a humanized-chimeric mouse model, thus providing a versatile vector which has the potential to be used in both preclinical testing and human clinical trials for both liver-based diseases and diabetes." @default.
- W2955925866 created "2019-07-12" @default.
- W2955925866 creator A5002156552 @default.
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- W2955925866 date "2019-06-27" @default.
- W2955925866 modified "2023-09-26" @default.
- W2955925866 title "Using a barcoded AAV capsid library to select for novel clinically relevant gene therapy vectors" @default.
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