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Gene Therapy

Study Results from University of Toronto in the Area of Gene Therapy Published

Published in Gene Therapy Weekly, March 15th, 2012

2012 MAR 15 -- Data detailed in "Gene therapy: light is finally in the tunnel" have been presented. According to the authors of recent research from Toronto, Canada, "After two decades of ups and downs, gene therapy has recently achieved a milestone in treating patients with Leber's congenital amaurosis (LCA). LCA is a group of inherited blinding diseases with retinal degeneration and severe vision loss in early infancy."

"Mutations in several genes, including RPE65, cause the disease. Using adeno-associated virus as a vector, three independent teams of investigators have recently shown that RPE65 can be delivered to retinal pigment epithelial cells of LCA...

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