Using a bar-coded AAV capsid library to select for novel clinically relevant gene therapy vectors
Katja Pekrun, Gustavo de Alencastro, Qing-Jun Luo, Jun Liu, Young‐Jin Kim, Sean C. Nygaard, Feorillo Galivo, Feijie Zhang, Ren Song, Matthew R. Tiffany, Jianpeng Xu, Matthias Hebrok, Markus Grompe, Mark A. Kay
The paper develops a barcoded AAV capsid library to identify novel gene therapy vectors with improved transduction efficiency in human islets and hepatocytes.
JCI Insight · Nov 13, 2019Peer-reviewedGenetics