Small Molecule Treatment Alleviates Photoreceptor Cilia Defects in LCA5-Deficient Human Retinal Organoids

Investigators reported the use of gene editing to generate isogenic LCA5 knock-out iPSC and their differentiation to retinal organoids. The molecular and cellular phenotype of the LCA5 KO retinal organoids was studied in detail and compared to isogenic controls as well as patient-derived retinal organoids.