Seeing is Believing: A Gene Therapy Success – Video

Posted: November 18, 2011 at 12:33 pm



Air date: Wednesday, December 15, 2010, 3:00:00 PM Timedisplayed is Eastern Time, Washington DC Local Category: Wednesday Afternoon Lectures Description: Gene therapy has the potential to reverse disease or prevent further deterioration of vision in patients with incurable inherited retinal degeneration. The demonstration of safe and stable recovery of retinal/visual function in 12 children and adults with Leber congenital amaurosis (LCA) due to RPE65 mutations in a trial being carried out at The Children's Hospital of Philadelphia (CHOP), and encouraging results from four other clinical trials for LCA-RPE65 provide great hope for people with other more common blinding diseases. This presentation will describe the animal studies that led to the clinical trials and the latest safety and efficacy results in the LCA-RPE65 clinical trial being held at CHOP

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Seeing is Believing: A Gene Therapy Success - Video

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