Recreating Pathophysiology of CLN2 Disease and Demonstrating Reversion by TPP1 Gene Therapy in hiPSC-Derived Retinal Organoids and Retina-on-Chip

Leveraging hiPSC-derived retinal organoids, retinal pigmented epithelial cells, and the retina-on-chip system, scientists established an in vitro neuronal ceroid lipofuscinosis type 2 (CLN2) model that recreates the principal histological hallmarks.