National Institutes of Health
Wednesday Afternoon Lecture Series

"Seeing is Believing: A Gene Therapy Success"
Dr. Jean Bennett
December 15
, 2010

Lecture Summary:
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. This Phase 1-2 study is now >3 years past initiation. In addition, it will describe some of the challenges presented by the nature of the targeted disease itself, hurdles that have been navigated in order to conduct the study, and issues of importance for eventual approval of gene augmentation as a therapy for LCA and other blinding diseases.

Lecture Objectives:
1. To understand the strengths and limitations of animal models of inherited retinal degeneration
2. To learn the status of gene therapy for congenital blindness
3. To become familiar with the strategies and challenges of gene based treatments for blindness


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