Preclinical Pharmacology and Toxicology Studies of an AAV8-CYP4V2 Gene Therapy VGR-R01 for the Treatment of Bietti Crystalline Dystrophy

In vitro, VGR-R01 induced a dose-dependent expression of the CYP4V2 protein, which significantly enhanced fatty acid hydroxylase activity and reduces lipid droplet accumulations in the retinal pigment epithelium cells.