Gene Therapy

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

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

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

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

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

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

Enzyme Replacement Therapy The human body utilizes enzymes to digest food and carry out vital activities for normal life function 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. 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 Some enzymes can be replaced by the delivery of a functioning gene

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

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

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