BioengineeringbioRxiv
EX VIVO GENE EDITING AND CELL THERAPY FOR HEREDITARY TYROSINEMIA TYPE 1
I. Ates, T. Rathbone, C. Stuart…·R. 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.
DOI 10.1097/hc9.0000000000000424