Gene Therapy

1. Gene Therapy

2. Gene Therapy • Gene Therapy is a technique for correcting defective genes responsible for disease development • Gene therapy is the process of introducing a new gene into a cell in order to replace one that isnt functioning correctly

3. Monoclonal antibodies • Hybridomas made of monoclonal antibodies and transformed cancer cells • Attack malignant specific tumor cells and prevent tumor growth by targeting specific cell receptors • Gene therapy can deliver these antibodies to specific tumor cells

4. Tissue Plasminogen Activator (tPA) • Protein used in the breakdown of blood clots • Converts plaminogen to plasmin • Use disabled adenovirus to insert gene into healthy aleg veins of rabbits. • Vein pumps out TPA • Use vein as a bypass for arterial blocks

5. Interferons • Cytokine produced by immune cells in response to foreign agents • Inhibit viral replication within cells • Activate natural killer T cells and macrophages • Increase antigen presentation and resistance of host cell to viral infection • Used to treat cancer because cells do not proliferate as easily • Has unfortunate short serum half-life • Use gene therapy to direct gene for interferon to tumor cells

6. Insulin • A protein hormone that aids in the storage of sugar in the blood • Insulin is produced in the pancreas • Diabetics do not make enough or faulty insulin • Diabetics must take insulin shots to regulate blood sugar levels • Can we insert gene into pancreas cells to get them to express insulin?

7. Human Growth Hormone • HGH produced in the pituitary gland • HGH causes bones, muscles and organs to grow • Too little leads to dwarfism • HGH is taken by injection • HGH gene therapy for growth, anti-aging affects, and cancer

8. Enzyme Replacement Therapy • The human body utilizes enzymes to digest food and carry out vital activities for normal life function • Replacing an enzyme in a person in which that specific enzyme is deficient or absent. this is done an IV infusion containing the enzyme. • Some enzymes can be replaced by the delivery of a functioning gene

9. Retroviruses • RNA virus • Target only actively dividing cells as targets • Higher risk of mutating the genome

10. Adenoviruses • Non dividing cells are infected with the virus • Not as pathogenic as retrovirus • Used for cystic fibrosis and other lung disorders • Small risk of mutation

11. Non-Viral Delivery Methods • Liposome transfer Transfer of therapeutic material using phospholipids molecules • Microinjection DNA is injected into the nucleus of an animal cell