Work

iPSC-derived retinal organoids: Biological insights and progress toward photoreceptor replacement therapy

Year: 2026

Type: review

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.

Source: Cell Reports Medicine

DOI: 10.1016/j.xcrm.2026.103053

Authors: Akiko Maeda, Xiaoxu Han, Michiko Mandai

Institutions: Ritsumeikan University, Kobe City Eye Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Peking Union Medical College Hospital, RIKEN Center for Biosystems Dynamics Research

Language: English

Topic: Retinal Development and Disorders

Subfield: Ophthalmology

Field: Medicine

Domain: Health Sciences

SDG: Good health and well-being

Open Access status: gold

Estimated APC paid: $5,620