Unit 5.6 Gene therapy

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  • GENE THERAPY
    • Replacing defective genes that cause inherited diseases
    • Method 1 Somatic cell therapy
      • Doesn't result in inheritance
      • Treatment of affected cells
      • Genes delivered via vectors
        • e.g. virus or liposome
    • Method 2 Germ line therapy
      • Gene replaced in egg/embryo
      • Results in inheritance
      • Advantages
        • Gene therapy is permanent
        • Antibody against vector not made
      • Disadvantages
        • Distruption of other genes in DNA - cancer
        • Unethical to experiment on human embryos
    • Cystic Fibrosis
      • Alveoli + pancreatic duct clogged with mucus
      • Caused by CFTR gene
        • controls production of Cl- channel protein
      • Cl- can't pass out of epithelial cells so less water moves out of the cells - mucus becomes thick
      • Inhaling vector using aerosol
        • Not permanent, only lungs treated
      • Screening
        • Sweat test
        • Blood test
        • Chorionic villus sampling/ Amniocentesis

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