Leber's congenital amaurosis (LCA) is an autosomal recessive eye disease. LCA results in eye disorders, including severe loss of vision, at birth. LCA has been successfully treated by gene therapy, using a virus instead of a plasmid as the vector. Adeno-associated virus (AAV) vectors containing the therapeutic allele were injected directly into the retina, the layer at the back of the eye containing the photoreceptor cells. People who had been blind from a young age were able to see again. There is a risk associated with the injection method used to deliver the vectors, as it might cause the retina to detach, damaging vision. This method of delivery was first used for LCA before being trialled on other retinal diseases that gradually reduce the vision of people as they get older.
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