AAV2-Mediated Gene Therapy for Bietti Crystalline Dystrophy Provides Functional CYP4V2 in Multiple Relevant Cell Models

Researchers demonstrated that HEK293, ARPE19, and patient induced pluripotent stem cell-derived RPE cells transduced with AAV2 vectors encoding codon optimization of CYP4V2 resulted in elevated protein expression levels of CYP4V2 compared to those transduced with AAV2 vectors encoding wild type.