Curative levels of endogenous gene replacement achieved in non-human primate liver using programmable genomic integration
Jenny Xie, Maike Thamsen Dunyak, Patrick Hanna, Angela Xinyi Nan, Brett Estes, Jesse C. Cochrane, Shuai Wu, Jie Wang, Connor McGinnis, Qiang Wang, Rejina Pokharel, Dev Paudel, Jason Zhang, Dan Li, Parth H. Amin, , Jonathan D. Finn
This paper demonstrates programmable genomic integration in non-human primate liver, achieving curative levels of gene expression in over 50% of liver cells.
bioRxiv · Oct 12, 2024 · PreprintBioengineering