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Success of gene therapy for a form of inherited blindness depends on timing

September 9, 2019

Using a canine model of the vision disorder Leber congenital amaurosis, Penn researchers found that photoreceptor cells continue to deteriorate after treatment if it is given too late.

With LCA Gene Therapy, a Rare Glimpse of the Adult Brain Adapting to New Experience

August 4, 2015

When people lose the ability to see, how do the visual parts of the brain change in response? And if they regain their sight, are the changes reversed?

NIH-funded Study Points Way Forward for Retinal Disease Gene Therapy

May 1, 2015

Gene therapy for Leber congenital amaurosis (LCA), an inherited disorder that causes vision loss starting in childhood, improved patients’ eyesight and the sensitivity of the retina within weeks of treatment.