Article: Gene Therapy Restores Sight to Children with Congenital Blindness.

Results from a breakthrough study, conducted by researchers at The Children's Hospital of Philadelphia (CHOP) and the University of Pennsylvania School of Medicine, show that gene therapy has restored significant vision in five children and seven adults who were previously blind. The Foundation Fighting Blindness funded the clinical trial and has been the long standing funding source of the research that made the trial possible. The study used gene therapy to treat participants with Leber's congenital amaurosis, a severe form of retinitis pigmentosa that causes blindness or substantial vision loss at birth. Trial participants demonstrated improvements in visual acuity, ...

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