Ex vivo therapeutic base and prime editing using chemically derived hepatic progenitors in a mouse model of tyrosinemia type 1
Y. Kim, J. Yu, S.-A. Hong, J. Eom, K. Jang, S.-N. Lee, J.-S. Woo, J. Jeong, S. Bae, D. Choi
This study corrects a disease-causing mutation in a mouse model of tyrosinemia type 1 using ex vivo base and prime editing with chemically derived hepatic progenitors, improving survival rates.
bioRxiv · Sep 14, 2020 · PreprintCell Biology