Transplantation of genome-edited iPS cells delivers therapeutic molecules in vivo
Induced pluripotent stem (iPS) cells have a great impact on biology and medicine, and they are expected to improve regenerative medicine. Since 2014 when a sheet of retinal pigment epithelial cells derived from iPS cells was transplanted into patients with age-related macular degeneration, clinical trials have been conducted with various cell types derived from iPS cells. While iPS cells derived from healthy individuals have been used so far, it is expected that transplantation therapy using iPS cells can be enhanced through genetic modification in the future.
Therefore, we addressed this possibility by utilizing a Fabry disease mouse model, ...

















