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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.
Seeing is believing : a gene therapy success [electronic resource] / Jean Bennett.
Series:
NIH director's Wednesday afternoon lecture
Author:
Bennett, Jean. National Institutes of Health (U.S.)
Publisher:
Other Title(s):
NIH director's Wednesday afternoon lecture
Abstract:
(CIT): 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.
Subjects:
Clinical Trials as Topic Dogs Genetic Therapy--methods Leber Congenital Amaurosis--therapy
