RPE65 Gene Therapy Shown Effective in Leber Congenital Amaurosis

January 2014

Five patients with LCA2 had improved visual function within a few months after treatment with a single unilateral injection of adeno-associated virus AAV2-hRPE65v2, and the effect remained stable for 3 years. Visual function measurements included best-corrected visual acuity, visual field, and reduction in nystagmus frequency compared with baseline.