Transplantation of Embryonic and Induced Pluripotent Stem Cell-Derived 3D Retinal Sheets into Retinal Degenerative Mice

• 3D retina was formed from ESCs/iPSCs using retinoic acid receptor antagonist
• ESC/iPSC retina developed a structured layer of photoreceptors in rd1 host mice
• Transplanted photoreceptors formed complete inner and outer segments
• Transplanted photoreceptors morphologically made synapses with host bipolar cells

SummaryIn this article, we show that mouse embryonic stem cell- or induced pluripotent stem cell-derived 3D retinal tissue developed a structured outer nuclear layer (ONL) with complete inner and outer segments even in an advanced retinal degeneration model (rd1) that lacked ONL. We also observed host-graft synaptic connections by immunohistochemistry. This study provides a “proof of concept” for retinal sheet transplantation therapy for advanced retinal degenerative diseases.

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