Lukas Schmidheini

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

This study uses prime editing in mice brains to introduce a mutation in the Adrb1 gene, leading to increased activity and exploratory behavior.

In vivo prime editing corrects phenylketonuria in mice by repairing the Pah gene mutation and reducing blood phenylalanine levels.