Tanja Rothgangl

The paper develops scOUT-seq to profile DNA repair outcomes in single cells and finds that repair outcomes vary significantly across cell types and subtypes.

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.

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

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.