Gene therapy can restore vision in LCA patients
In a clinical trial at The Children’s Hospital of Philadelphia, researchers from The University of Pennsylvania have used gene therapy to safely restore vision to a significant degree in three young adults with a rare, inherited blinding disease, Leber congenital amaurosis (LCA). The LCA gene therapy performed through a surgical procedure reflected no signs of causing inflammation in the retina or other toxic side effects. The innovative treatment might be effective in treating possibly other congenial retinal diseases.
