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Updated: Sep 27, 2026

Generation of Retinal Organoids from Healthy and Retinal Disease-Specific Human-Induced Pluripotent Stem Cells
Published on: December 9, 2022
iPSC-derived retinal organoids: Biological insights and progress toward photoreceptor replacement therapy
Akiko Maeda1, Xiaoxu Han2, Michiko Mandai3
1Research Center, Kobe City Eye Hospital, Hyogo 650-0047, Japan; Research Organization of Science and Technology, Ritsumeikan University, Shiga 525-8577, Japan.
Abstract:
Induced pluripotent stem cell (iPSC)-derived retinal organoids (ROs) have emerged as a platform for photoreceptor replacement therapy, supported by two complementary approaches: cone precursor cell transplantation and RO transplantation. Both have demonstrated proof of concept for functional synaptic integration with the host retinal network, with the first clinical application of iPSC-derived RO transplantation conducted in 2020. Key mechanistic insights include the role of the host environment in cone integration, genome editing-based enhancement of host-graft synaptogenesis recapitulating developmental synaptic assembly, and synaptic rewiring capacity in the adult primate retina. Beyond biology, successful clinical translation requires establishing meaningful endpoints that reflect stepwise therapeutic improvement, scalable good manufacturing practice (GMP)-compliant manufacturing including validated preservation protocols, and regulatory frameworks accommodating progenitor-stage cell therapies. Together, these advances offer a realistic path toward vision restoration in patients with currently untreatable retinal degenerative diseases.
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