Artificial Intelligence as a Catalyst for Cell-Based Therapies in Retinal Disease

Abstract

Retinal degenerative diseases, including age-related macular degeneration (AMD), retinitis pigmentosa (RP), and Stargardt disease, represent leading causes of irreversible vision loss worldwide, collectively affecting hundreds of millions of people. Cell-based therapies, particularly those leveraging induced pluripotent stem cells (iPSCs), retinal pigment epithelium (RPE) transplantation, and CRISPR-Cas9 gene editing, have emerged as promising treatment modalities that may preserve, replace, or partially restore retinal function. However, these approaches face significant challenges in patient stratification, manufacturing consistency, surgical precision, and long-term outcome monitoring. Artificial intelligence (AI), encompassing machine learning (ML), deep learning (DL), and computer vision, is increasingly being integrated across the cell therapy pipeline to address these limitations. This review comprehensively explores the role of AI in enhancing cell-based retinal therapies, spanning disease diagnosis and patient selection, stem cell quality control and differentiation optimization, CRISPR off-target detection, retinal organoid characterization, scalable manufacturing, and post-transplantation monitoring through multimodal retinal imaging. Throughout, we distinguish direct retinal cell-therapy evidence from retinal-adjacent, cross-domain, and conceptual evidence. We also propose an AI-powered end-to-end pipeline that connects patient profiling through gene editing, cell production, and transplantation to longitudinal outcomes tracking (a forward-looking framework that needs to be clinically validated in future). While AI offers powerful tools to enhance efficacy, reproducibility, and personalization, it does not replace the clinical and biological foundations of cell therapy. Critical challenges remain, including data scarcity, algorithmic interpretability, regulatory compliance, and clinical validation. This review concludes that the strategic integration of AI has plausible potential to become supportive infrastructure for scalable and reproducible cell-based treatments for retinal diseases, although its necessity has not been demonstrated and prospective clinical validation is still required, and biological insight and rigorous empirical verification remain fundamental.