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- Leber congenital amaurosis (LCA) is a rare genetic eye disease that appears at birth or in the first few months of life.

- It's caused by a mutation in a gene, such as RPE65, which is important for vision.

- Luxturna is an (FDA approved) gene therapy.

- This AAV2 vector delivers a functional copy of the RPE65 gene to the photoreceptor cells that need it, stopping symptoms getting worse!

 

 

 

Find out more about the work of the Gill Hyde Gene Medicine research group at the University of Oxford.