BioengineeringbioRxivPreprint
Treatment of a genetic liver disease in mice through transient prime editor expression
T. Rothgangl, E. I. Ioannidi, Y. Weber…·G. Schwank
This paper demonstrates transient prime editor expression in mice to correct a genetic liver disease, reducing blood phenylalanine levels below the therapeutic threshold.
DOI 10.1101/2024.01.22.575834