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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!



This is one example of how researchers are taking advantage of adeno-associated virus (AAV) vectors to treat diseases.

 Adeno-associated virus (AAV) vectors:

  • Efficient targeted delivery in vivo: many different serotypes (e.g. AAV2 used here) with different tissue or cell-type tropism.
  • Parent virus is not pathogenic in humans.
  • Long-term expression from long-lived cells.
  • Relatively easy to produce high quantities.


 Click here to discover how an AAV9 vector is also being used to cure paralysis!



Other viral vectors are also being used or developed to treat disease: 




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