Treatment of a genetic liver disease in mice through transient prime editor expression
T. Rothgangl, E. I. Ioannidi, Y. Weber, A. Talas, D. Boeck, M. Matsushita, E. A. Villiger, L. Schmidheini, J. Moon, P. J. C. Lin, S. H. Y. Fan, K. F. Marquart, C. Schwerdel, N. Rimann, E. Faccin, , 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.
bioRxiv · Jan 22, 2024 · PreprintBioengineering