Stem cell therapy for inherited retinal diseases: Trends and insights from 2000 to 2024

Stem cell therapy has emerged as a promising strategy for inherited retinal diseases (IRDs), yet its global research trajectory has not been systematically mapped. This study analyzes publication trends, leading contributors, and thematic evolution of IRD stem cell therapy research from 2000 to 2024. Publications were retrieved from the Web of Science Core Collection and analyzed with CiteSpace and VOSviewer. We identified 1060 articles with a steady rise in annual output. The United States and China were the most prolific countries; University College London and the University of Iowa were major institutions; and key outlets included Stem Cell Research & Therapy, Investigative Ophthalmology & Visual Science, and Cell Transplantation. Keyword and co-citation analyses reveal a clear trajectory: early emphasis on stem cell-derived retinal pigment epithelium transplantation for photoreceptor rescue, subsequent expansion to photoreceptor precursor and retinal organoid replacement, and recent movement toward early clinical translation. Persisting challenges include long-term graft survival, functional integration, and immune compatibility. Overall, this bibliometric roadmap clarifies how the field is transitioning from foundational studies to translational application and highlights priorities for interdisciplinary collaboration to accelerate clinical advancement.