Gene TherapyGene Therapy

Gene TherapyGene Therapy

Gene Therapy is a technique for Gene Therapy is a technique for correcting defective genes correcting defective genes responsible for disease responsible for disease developmentdevelopment

Gene therapy is the process of Gene therapy is the process of introducing a new gene into a cell introducing a new gene into a cell in order to replace one that isnt in order to replace one that isnt functioning correctlyfunctioning correctly

Monoclonal antibodiesMonoclonal antibodies

Hybridomas made of monoclonal Hybridomas made of monoclonal antibodies and transformed cancer antibodies and transformed cancer cellscells

Attack malignant specific tumor Attack malignant specific tumor cells and prevent tumor growth by cells and prevent tumor growth by targeting specific cell receptorstargeting specific cell receptors

Gene therapy can deliver these Gene therapy can deliver these antibodies to specific tumor cellsantibodies to specific tumor cells

Tissue Plasminogen Activator Tissue Plasminogen Activator (tPA)(tPA)

Protein used in the breakdown of blood Protein used in the breakdown of blood clotsclots

Converts plaminogen to plasminConverts plaminogen to plasmin Use disabled adenovirus to insert gene Use disabled adenovirus to insert gene

into healthy aleg veins of rabbits.into healthy aleg veins of rabbits. Vein pumps out TPAVein pumps out TPA Use vein as a bypass for arterial blocksUse vein as a bypass for arterial blocks

InterferonsInterferons

Cytokine produced by immune cells in Cytokine produced by immune cells in response to foreign agentsresponse to foreign agents

Inhibit viral replication within cellsInhibit viral replication within cells Activate natural killer T cells and Activate natural killer T cells and

macrophagesmacrophages Increase antigen presentation and Increase antigen presentation and

resistance of host cell to viral infectionresistance of host cell to viral infection Used to treat cancer because cells do not Used to treat cancer because cells do not

proliferate as easilyproliferate as easily Has unfortunate short serum half-lifeHas unfortunate short serum half-life Use gene therapy to direct gene for Use gene therapy to direct gene for

interferon to tumor cellsinterferon to tumor cells

InsulinInsulin A protein hormone that aids in the A protein hormone that aids in the

storage of sugar in the bloodstorage of sugar in the blood Insulin is produced in the pancreasInsulin is produced in the pancreas Diabetics do not make enough or Diabetics do not make enough or

faulty insulinfaulty insulin Diabetics must take insulin shots to Diabetics must take insulin shots to

regulate blood sugar levelsregulate blood sugar levels Can we insert gene into pancreas Can we insert gene into pancreas

cells to get them to express insulin?cells to get them to express insulin?

Human Growth Human Growth HormoneHormone HGH produced in the pituitary HGH produced in the pituitary

glandgland HGH causes bones, muscles and HGH causes bones, muscles and

organs to groworgans to grow Too little leads to dwarfismToo little leads to dwarfism HGH is taken by injectionHGH is taken by injection HGH gene therapy for growth, anti-HGH gene therapy for growth, anti-

aging affects, and canceraging affects, and cancer

Enzyme Replacement Enzyme Replacement TherapyTherapy The human body utilizes enzymes The human body utilizes enzymes

to digest food and carry out vital to digest food and carry out vital activities for normal life functionactivities for normal life function

Replacing an enzyme in a person in Replacing an enzyme in a person in which that specific enzyme is which that specific enzyme is deficient or absent. this is done an deficient or absent. this is done an IV infusion containing the enzyme.IV infusion containing the enzyme.

Some enzymes can be replaced by Some enzymes can be replaced by the delivery of a functioning genethe delivery of a functioning gene

RetrovirusesRetroviruses

• RNA virusRNA virus• Target only actively dividing Target only actively dividing

cells as targetscells as targets• Higher risk of mutating the Higher risk of mutating the

genomegenome

AdenovirusesAdenoviruses

Non dividing cells are infected Non dividing cells are infected with the viruswith the virus

Not as pathogenic as retrovirusNot as pathogenic as retrovirus Used for cystic fibrosis and other Used for cystic fibrosis and other

lung disorderslung disorders Small risk of mutationSmall risk of mutation

Non-Viral Delivery Non-Viral Delivery MethodsMethods Liposome transferLiposome transfer

Transfer of therapeutic material using Transfer of therapeutic material using phospholipids moleculesphospholipids molecules

MicroinjectionMicroinjectionDNA is injected into the nucleus of an DNA is injected into the nucleus of an

animal cellanimal cell