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Showing 1-6 of 6 Resources
  • 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.

  • Origin of a Gene Mutation Causing Early-Onset Alzheimer’s Disease

    Origin of a Gene Mutation Causing Early-Onset Alzheimer’s Disease

    Data Point

    A team of scientists built a pedigree of a large extended family in Antioquia, Colombia, that has a high prevalence of early-onset familial Alzheimer’s disease due to a mutation in the PSEN1 gene. Worksheets also available in Spanish.

  • Identifying Autism Genes by Tracking Gene Mutations

    Identifying Autism Genes by Tracking Gene Mutations

    Data Point

    A team of scientists used homozygosity mapping to map SNPs on chromosome 3 in a family with an autistic son to identify a likely gene that caused the disorder.