In Vivo Delivery of Therapeutic Molecules by Transplantation of Genome-Edited Induced Pluripotent Stem Cells
I. Nakajima, T. Tsukimura, T. Ono, T. Shiga, H. Shitara, T. Togawa, H. Sakuraba, Y. Miyaoka
This study shows genome-edited iPSCs can secrete therapeutic molecules to treat Fabry disease in mice.
bioRxiv · Jan 3, 2023 · PreprintCell Biology