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Genes As Medicine

17 minutes 16 seconds

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Illustration of a sphere with an uneven surface. Caption: Scientists strip away the virus's harmful genes

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: 17 minutes 16 seconds

Lab equipment in background with illustration of DNA strand, question marks, and an arrow pointing to the horn on a unicorn. Caption: We'd want to know what parts of the genome make the horn.
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Episode 2
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Grade Level: 9 - 12
Lab equipment in background with illustration of connected neurons. Caption: glia activate and help fix the neurons
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Grade Level: 8 - 12
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Grade Level: 10 - 12
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Grade Level: 4 - 8
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Grade Level: 3 - 5
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Grade Level: 5 - 10
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