hPSC-based treatment of retinal diseases - Current progress and challenges

Area of Science:

• Ophthalmology
• Regenerative Medicine
• Cell Biology

Background:

• Degenerative retinal diseases, including age-related macular degeneration (AMD) and inherited retinal diseases (IRDs), lead to irreversible vision loss due to damage to retinal pigment epithelium (RPE) and photoreceptor cells (PRCs).
• Current therapeutic options for these conditions are limited, necessitating the exploration of novel treatment strategies.

Purpose of the Study:

• To review recent advancements in cell-based transplantation therapies for RPE and PRC replacement in degenerative retinal diseases.
• To highlight the progress and outcomes of clinical studies involving different RPE and PRC transplantation formulations.
• To discuss novel approaches, including genome-edited retinal organoid sheets, aimed at enhancing functional integration of transplanted cells.

Main Methods:

• Review of clinical studies on RPE transplantation using sheets, suspensions, and strips.
• Analysis of progress in photoreceptor cell (PRC) transplantation, from fetal retinal grafts to current research.
• Examination of recent clinical work involving retinal sheet transplantation and genome-edited retinal organoid sheets.

Main Results:

• Clinical studies have demonstrated the safety and potential efficacy of various RPE transplantation formulations.
• Progress in PRC transplantation has advanced from early fetal grafts to more sophisticated cell delivery methods.
• Genome-edited retinal organoid sheets show potential for improved functional integration by modulating graft composition.

Conclusions:

• Cell-based therapies, including RPE and PRC transplantation, represent a promising avenue for treating degenerative retinal diseases.
• Ongoing innovations in transplantation techniques and cell engineering are crucial for improving visual function restoration.
• The established safety profile of regenerative cell-based therapies supports continued exploration of their future clinical applications.