Treatment of a metabolic liver disease by in vivo prime editing in mice
Desirée Böck, Tanja Rothgangl, Lukas Villiger, Lukas Schmidheini, Nicholas Mathis, Eleonora I. Ioannidi, Susanne Kreutzer, Zacharias Kontarakis, Nicole Rimann, Hiu Man Grisch‐Chan, Beat Thöny, Gerald Schwank
In vivo prime editing corrects phenylketonuria in mice by repairing the Pah gene mutation and reducing blood phenylalanine levels.
bioRxiv · Aug 17, 2021 · PreprintGenetics