Sustained safety, efficacy of gene therapy for inherited retinal disease
Monday, September 19, 2011 - 09:31
in Health & Medicine
Three years ago, preliminary but encouraging results were announced regarding the safety and success of gene therapy in a small cohort of patients with an inherited form of blindness known as Leber congenital amaurosis (LCA). Now, researchers at the Perelman School of Medicine at the University of Pennsylvania and the Scheie Eye Institute, have taken the next major step in their pursuit of treatment for this previously incurable vision loss by showing continued safety and efficacy of gene therapy in a larger and age-diverse group of LCA patients.