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Updated: Jun 11, 2025
Transpupillary-Guided Trans-Scleral Transplantation of Subretinal Grafts in a Retinal Degeneration Mouse Model
Published on: January 26, 2024
Graft survival of major histocompatibility complex deficient stem cell-derived retinal cells
Masaaki Ishida1,2, Tomohiro Masuda1,3,4, Noriko Sakai1,3
1Laboratory for Retinal Regeneration, Center for Developmental Biology, RIKEN, Kobe, Japan.
Background:
Gene editing of immunomodulating molecules is a potential transplantation strategy to control immune rejection. As we noticed the successful transplantation of retinal pigment epithelium (RPE) derived from embryonic stem cells of a cynomolgus monkey that accidentally lacked MHC class II (MHC-II) molecules, we hypothesized immune rejection could be evaded by suppressing MHC-II.
Methods:
Gene editing by the Crispr/Cas9 system was performed in induced pluripotent stem cells derived from a cynomolgus monkey (miPSCs) for targeted deletion of the gene coding class II MHC trans-activator (CIITA). Then the CIITA-knocked out miPSCs were differentiated into RPE cells to generate miPSC-derived MHC-II knockout RPE. The MHC-II knockout or wild-type RPEs were transplanted into the eyes of healthy cynomolgus monkeys. All monkeys used in this study were male.
Results:
Here we show when MHC-II knockout RPE are transplanted into monkey eyes, they show suppressed immunogenicity with no infiltration of inflammatory cells, leading to successful engraftment.
Conclusions:
Our results reasonably evidence the efficacy of MHC-II knockout iPSC-RPE transplants for clinical application.
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