Cell BiologybioRxivPreprint
In Vivo Delivery of Therapeutic Molecules by Transplantation of Genome-Edited Induced Pluripotent Stem Cells
I. Nakajima, T. Tsukimura, T. Ono…·Y. Miyaoka
This study shows genome-edited iPSCs can secrete therapeutic molecules to treat Fabry disease in mice.
DOI 10.1101/2023.01.03.522057