iPSC-RPE / Beijing Tongren Hospital - LARVOL DELTA

Wnt signaling dysregulation drives Stargardt disease pathogenesis. (PubMed, Sci China Life Sci) - "Using patient-derived iPSC-RPE harboring distinct ABCA4 mutations, we demonstrate that ABCA4 loss disrupts RPE hexagonal morphology, reduces pigmentation, and aberrantly activates neural retinal differentiation...Our work positions non-canonical Wnt agonism as a therapeutic strategy for Stargardt and underscores the broader imperative for mutation-tailored interventions in inherited retinal diseases. By revealing phenotypic convergence on Wnt signaling dysregulation in ABCA4-deficient RPE, this study provides a framework for understanding epithelial-pathway-driven degeneration, with implications for AMD, fibrosis, and developmental disorders characterized by polarity loss."

Journal • Age-related Macular Degeneration • CNS Disorders • Developmental Disorders • Fibrosis • Immunology • Inherited Retinal Dystrophy • Macular Degeneration • Ophthalmology • Psychiatry • Retinal Disorders

Safety and Efficacy of Autologous Transplantation of iPSC-RPE in the Treatment of Macular Degeneration (clinicaltrials.gov) - P1 | N=10 | Recruiting | Sponsor: Beijing Tongren Hospital

New P1 trial • Age-related Macular Degeneration • Macular Degeneration • Ophthalmology • Retinal Disorders • Transplantation • Wet Age-related Macular Degeneration