Cell-based regenerative therapy for retinal diseases: challenges and emerging bioengineering strategies

Area of Science:

• Ophthalmology
• Regenerative Medicine
• Cell Biology

Background:

• Retinal diseases cause irreversible photoreceptor cell (PRC) loss, leading to vision impairment.
• Gene therapy is limited in advanced stages and doesn't replace lost PRCs.
• Cell-based regenerative therapy aims to restore vision by rebuilding retinal circuitry.

Purpose of the Study:

• To review cell-based regenerative approaches for retinal diseases.
• To outline advantages and limitations of cell suspensions vs. retinal sheets.
• To discuss challenges and solutions for clinical translation.

Main Methods:

• Review of preclinical and clinical evidence for cell suspension and retinal sheet transplantation.
• Analysis of a first-in-human trial of iPSC-derived retinal organoid sheets.
• Discussion of bioengineering and biological solutions for transplantation.

Main Results:

• Cell-based therapies, including retinal organoid sheets, show potential for PRC replacement.
• A first-in-human trial in retinitis pigmentosa demonstrated safety and two-year graft survival.
• Advancements in developmental biology and genome engineering are accelerating progress.

Conclusions:

• Cell-based regenerative medicine is transitioning to clinical exploration for retinal diseases.
• Transplantation strategies may extend beyond retinitis pigmentosa to macular diseases.
• Functional vision restoration is becoming increasingly achievable through these approaches.