Kim Fabiano Marquart

This paper demonstrates transient prime editor expression in mice to correct a genetic liver disease, reducing blood phenylalanine levels below the therapeutic threshold.

The paper describes a minimally invasive method for delivering AAV vectors to pig livers but finds low vector retention and no detectable editing in vivo.

This paper develops BE-DICT, a deep learning model trained on high-throughput screens to predict base editing outcomes with high accuracy.