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  • Cystic Fibrosis Mechanism and Treatment

    Cystic Fibrosis Mechanism and Treatment


    (2 min 31 sec) Mutations in the CFTR gene, which encodes a transmembrane ion channel, cause mucus buildup in the airways of patients with cystic fibrosis.

  • CRISPR-Cas 9 Mechanism & Applications

    CRISPR-Cas 9 Mechanism & Applications

    Click & Learn

    Explore this dynamic interactive to learn the basic principles of how this revolutionary tool works and the many ways in which scientists are using it in their research.

  • Film Guide for Genes as Medicine

    Film Guide for Genes as Medicine

    Film Guide

    The following classroom-ready resources complement the film Genes as Medicine. It tells the story of the successes and challenges that led to the development of the first FDA-approved gene therapy for a genetic disease.

  • The Search for a Mutated Gene

    The Search for a Mutated Gene

    Scientists at Work

    (8 min 54 sec) How do you identify the disease-causing mutation in a patient with an inherited disease? This video illustrates the steps involved—testing for genes that are the “usual suspects,” searching for mutations in novel genes, and assessing links between genotype and phenotype.

  • Genes as Medicine

    Genes as Medicine

    Short Film

    (17 min 16 sec) 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.

  • Central Dogma and Genetic Medicine

    Central Dogma and Genetic Medicine

    Click & Learn

    This interactive uses the central dogma as a model for exploring how modern molecular biology technologies can be used to treat genetic diseases.

  • Cardiology Virtual Lab

    Cardiology Virtual Lab

    Virtual Lab

    NEW! HTML 5 support added! This virtual lab will familiarize you with heritable diseases of the heart. Learn about the diagnostic tools used to examine and diagnose patients.