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    Gene therapy improves functional vision in Leber congenital amaurosis

    Las Vegas—A phase III gene therapy trial for Leber congenital amaurosis showed that patients’ mean mobility test scores promptly improved after treatment, and the benefit was sustained at 1 year, said Albert M. Maguire, MD.

    Treatment with adeno-associated viral vector to deliver the gene for human RPE65 (AAV2-hRPE65v2; SPK-RPE65) into the retinal pigment epithelium in patients with RPE65 mutation-associated inherited retinal dystrophies was safe and associated with highly statistically significant improvement in the primary endpoint assessing ambulatory vision, according to the results of the study.

    “This is the first randomized, controlled phase III gene therapy trial for a genetic disease of any kind,” said Dr. Maguire, at Retina 2015 during the annual meeting of the American Academy of Ophthalmology.

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