Watch the story of how gene therapy restored the sight of a nearly-blind young patient. Told from the perspective of two researchers who spent over 25 years working to develop this breakthrough technology, this short film chronicles their successes and challenges, and illustrates how the method works to treat inherited conditions.
Gene therapy is a method for treating inherited diseases by delivering corrective versions of genes to patients. The development of this technology has been on a roller coaster of advances and setbacks over the last two decades. Few researchers are more familiar with that roller coaster than Dr. Jean Bennett and Dr. Albert Maguire at the University of Pennsylvania. They have focused their careers on developing a successful gene therapy for an inherited form of childhood blindness called Leber Congenital Amaurosis (LCA). On October 12, 2017, science advisors to the U.S. Food and Drug Administration (FDA) unanimously agreed that the benefits of this gene therapy far outweigh the possible risks. If approved by the FDA, the LCA treatment will become the first gene therapy available to treat an inherited disease.
This film tells the story of how the LCA gene therapy was developed. Students will learn how autosomal recessive conditions are inherited, how scientists can use modified viruses to deliver human genes to cells, what makes the eye an ideal tissue for gene therapy, and how model organisms are used to test treatments before they are tested in patients.
5 окт 2024
