iPSC-derived retinal organoids: Biological insights and progress toward photoreceptor replacement therapy
4 affiliations
- 1Research Center, Kobe City Eye Hospital, Hyogo 650-0047, Japan
- 2Research Organization of Science and Technology, Ritsumeikan University, Shiga 525-8577, Japan
- 3Department of Ophthalmology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Beijing 100730, China
- 4RIKEN Center for Biosystems Dynamics Research, Hyogo 650-0047, 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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BibTeX
@article{maeda2026ipscderived,
title = {{iPSC-derived retinal organoids: Biological insights and progress toward photoreceptor replacement therapy}},
author = {Akiko Maeda and Xiaoxu Han and Michiko Mandai},
journal = {Cell Reports Medicine},
year = {2026},
month = sep,
doi = {10.1016/j.xcrm.2026.103053},
url = {https://doi.org/10.1016/j.xcrm.2026.103053},
note = {CC BY 4.0},
}
RIS
TY - JOUR
TI - iPSC-derived retinal organoids: Biological insights and progress toward photoreceptor replacement therapy
AU - Akiko Maeda
AU - Xiaoxu Han
AU - Michiko Mandai
PY - 2026
DA - 2026/09/25
JO - Cell Reports Medicine
DO - 10.1016/j.xcrm.2026.103053
UR - https://doi.org/10.1016/j.xcrm.2026.103053
AB - 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.
SN - 2666-3791
ER -