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Genes As Medicine

18 minutes

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Gene therapy is a method for treating inherited diseases by delivering corrective versions of genes to patients. Dr. Jean Bennett and Dr. Albert Maguire focused their careers on developing a successful gene therapy for an inherited form of childhood blindness called Leber congenital amaurosis (LCA). This documentary 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.

Media Details

Runtime: 18 minutes

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