AAV2-mediated gene therapy for Bietti crystalline dystrophy provides functional CYP4V2 in multiple relevant cell models

AAV2-mediated gene therapy for Bietti crystalline dystrophy provides functional CYP4V2 in multiple relevant cell models

Abstract 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 s...

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Bibliographic Details
Main Authors: Jiang-Hui Wang, Grace E. Lidgerwood, Maciej Daniszewski, Monica L. Hu, Georgina E. Roberts, Raymond C. B. Wong, Sandy S. C. Hung, Michelle E. McClements, Alex W. Hewitt, Alice Pébay, Doron G. Hickey, Thomas L. Edwards
Format: Article
Language:English
Published: Nature Portfolio 2022-06-01
Series:Scientific Reports
Online Access:https://doi.org/10.1038/s41598-022-12210-8

Internet

https://doi.org/10.1038/s41598-022-12210-8

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