RPE65 Gene Therapy Shown Effective in Leber Congenital Amaurosis
- Jan 2, 2014
- 1 min read
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.
