Genes as Medicine
This film describes the scientific principles and the research efforts involved in the development of a gene therapy for a congenital form of blindness, and how a young patient benefited from this medical breakthrough.
Gene therapy is an approach for treating inherited diseases by delivering corrective versions of genes to patients. Dr. Jean Bennett and Dr. Albert Maguire at the University of Pennsylvania have focused their careers on developing a successful gene therapy for an inherited form of childhood blindness called Leber congenital amaurosis. This film tells the story of their successes and setbacks, while exploring how autosomal recessive conditions are inherited, how scientists use viruses to deliver human genes to cells, and how model organisms are used to test treatments before they are used in patients.
The “Abbreviated Film Guide” provides a short summary of the film, along with key concepts and connections to curriculum standards.
An audio descriptive version of the film is available via our media player.
gene therapy, Leber congenital amaurosis, viral delivery
Russell, Stephen, Jean Bennett, Jennifer A. Wellman, Daniel C. Chung, Zi-Fan Yu, Amy Tillman, Janet Wittes, et al. “Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial.” The Lancet 390 (2017): 849–860. https://doi.org/10.1016/S0140-6736(17)31868-8.
HS-LS1.A, HS-LS3.A, HS-LS3.B
IST-1, IST-2, IST-3
3.4, 3.5, B.4