The combination of the human iPSC platform with the recently developed gene editing and 3D organoid technologies could make human iPSCs an even more powerful cellular resource for stem cell-based cell therapy development. As a proof-of-principle, mouse iPSCs corrected through gene editing have been used to generate hematopoietic progenitors for successful treatment of sickle cell anemia in a mouse model174. Furthermore, the integration of genetically corrected human iPSCs with 3D organoids could allow tissues to be generated as sources for organ replacement therapies97. Indeed, human iPSC-derived liver organoids have been shown to successfully generate functional human liver-like tissues in transplanted mice in a proof-of-principle study99. However, there are still challenges to overcome for such approaches to become applicable in human cell therapy. For example, the potential off-target effects associated with gene editing need to be addressed, as do the limitations of organoids, as described in the section of “iPSC-based disease modeling”.
