Unit 5.6 Gene therapy
- Created by: theviahsiva
- Created on: 08-05-15 11:13
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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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