Article
AAV2-mediated gene therapy for Bietti crystalline dystrophy provides functional CYP4V2 in multiple relevant cell models.
Scientific reports - 9 Jun 2022
Wang Jiang-Hui, Lidgerwood Grace E, Daniszewski Maciej, Hu Monica L, Roberts Georgina E, Wong Raymond C B, Hung Sandy S C, McClements Michelle E, Hewitt Alex W, Pébay Alice, Hickey Doron G, Edwards Thomas L
Abstract excerpt
Bietti crystalline dystrophy (BCD) is an inherited retinal disease (IRD) caused by mutations in the CYP4V2 gene. It is a relatively common cause of IRD in east Asia. A number of features of this disease make it highly amenable to gene supplementation therapy. This study aims to validate a series of essential precursor in vitro experiments prior to developing a clinical gene therapy for BCD. We demonstrated that...
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