Effective AAV-Mediated Gene Replacement Therapy in Retinal Organoids Modelling AIPL1-Associated LCA4

Researchers investigated the potential of adeno-associated virus-mediated aryl hydrocarbon receptor interacting protein-like 1 (AIPL1) gene replacement therapy in two previously validated human retinal organoid models of Leber congenital amaurosis subtype 4 (LCA4).