Transcript Gene Therapy Gene Therapy

Gene Therapy
Gene Therapy
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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
Monoclonal antibodies
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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
Tissue Plasminogen Activator (tPA)
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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
Interferons
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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
Insulin
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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?
Human Growth Hormone
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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, antiaging affects, and cancer
Enzyme Replacement
Therapy
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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
Retroviruses
• RNA virus
• Target only actively dividing cells as
targets
• Higher risk of mutating the genome
Adenoviruses
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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
Non-Viral Delivery
Methods
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Liposome transfer
Transfer of therapeutic material using
phospholipids molecules
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Microinjection
DNA is injected into the nucleus of an animal
cell