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

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...

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Detalles Bibliográficos
Autores principales: 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.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2022
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9184470/
https://www.ncbi.nlm.nih.gov/pubmed/35680963
http://dx.doi.org/10.1038/s41598-022-12210-8

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9184470/
https://www.ncbi.nlm.nih.gov/pubmed/35680963
http://dx.doi.org/10.1038/s41598-022-12210-8

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