BioengineeringHepatology CommunicationsPeer-reviewed
EX VIVO GENE EDITING AND CELL THERAPY FOR HEREDITARY TYROSINEMIA TYPE 1
Ilayda Ates, Callie Stuart, Tanner Rathbone…·Renee N. Cottle
This study develops an ex vivo CRISPR gene editing protocol for hepatocytes to treat hereditary tyrosinemia type 1, showing successful engraftment and disease correction in mice.