During the last decade, a number of advances have been made in the area of gene therapy. In December 2017, FDA approved the first gene therapy product for an inherited disease. This drug can be used to treat patient suffering from Leber congenital amaurosis type 2 (LCA2), a disease that has no effective treatment. Children born with LCA2 have poor eyesight which gets progressively worse, with most children losing their vision completely before adulthood. The disease is caused by loss-of-function mutations in the in the RPE65 gene, which the drug Luxturna can compensate for by introducing a functional copy of the gene administered through an adeno-associated virus (AAV)-based vector.