The presence of a dose-dependent inflammatory response to AAV2-mediated retinal gene therapy was first observed in humans in a phase 1/2 clinical trial treating RPE65-associated Leber congenital amaurosis where five of the eight high dose patients (1 × 1012 vg/eye) had signs of intraocular inflammation, such as anterior uveitis, mild vitritis, and optic disc swelling [15]. Here, RPE65 is linked to Leber congenital amaurosis.