EX VIVO GENE EDITING AND CELL THERAPY FOR HEREDITARY TYROSINEMIA TYPE 1
I. Ates, T. Rathbone, C. Stuart, M. Barzi, G. He, A. M. Major, S. Srinivasan, A. B. Farris, K.-D. Bissig, 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.
bioRxiv · Sep 4, 2023Bioengineering