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

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...

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發表在:JCI Insight
Main Authors: Pekrun, Katja, De Alencastro, Gustavo, Luo, Qing-Jun, Liu, Jun, Kim, Youngjin, Nygaard, Sean, Galivo, Feorillo, Zhang, Feijie, Song, Ren, Tiffany, Matthew R., Xu, Jianpeng, Hebrok, Matthias, Grompe, Markus, Kay, Mark A.
格式: Artigo
語言:Inglês
出版: American Society for Clinical Investigation 2019
主題:
Technical Advance
在線閱讀:https://ncbi.nlm.nih.gov/pmc/articles/PMC6948855/
https://ncbi.nlm.nih.gov/pubmed/31723052
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1172/jci.insight.131610
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