Beat Thöny

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

This paper describes a gene therapy approach using liver cell transplantation to treat PKU in mice by modifying hepatocytes to resist paracetamol and replace diseased cells.

The paper evaluates AAV capsids for liver gene therapy in a pig xenograft model, identifying efficient capsids for transgene expression in porcine hepatocytes.

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.

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