Overall view of gene therapy

1. Kosar Rahimi
December 2017
Sharif University of Technology
Department of Chemical and Petroleum
Engineering

2. What are genes?
Units of inheritance
Encoding instructions on how to
make proteins
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3. What is gene therapy?
Gene therapy is an experimental technique that uses genes to treat
or prevent disease. (instead of using drugs or surgery)
 Inactivating, or “knocking out,” a mutated gene that is functioning
improperly.
 Introducing a new gene into the body to help fight a disease.
 Replacing a mutated gene that causes disease with a healthy
copy of the gene.
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4. Overall view on Replacing
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5. Types of gene therapy depend on target
Somatic gene therapy
• body
Germ line gene therapy
• Egg and sperm
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6. Types of somatic gene therapy
Ex vivo
Cells are modified out
outside the body and
then transplanted back
in again.
In vivo
Cells are changed in
cells when the cells are
still in the body.
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7. Ex vivo gene therapy
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8. in vivo gene therapy
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9. Vectors in gene therapy
 Vectors are vehicles of gene delivery into target cells.
 2 main classes:
o Viral vectors
o Non viral vectors
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10. Viral vectors
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11. Why Non viral vectors?
• unexpected
immune response
• oncogenic effects
problems
occurring
with viral
vectors
Non viral
vectors
Physical
method
Chemical
method
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12. Non viral vectors-physical approaches
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Carrier-free gene delivery
Naked DNA delivery

13. Non viral vectors-chemical approaches
categorization of chemical carrier
protecting the DNA
target delivery to specific cell types
increasing delivery of DNA to the cytosol or nucleus
controlled released of DNA
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14. Disadvantages of gene therapy
Very high cost
Having potential to give rise in other disorders
Ethical issues
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15. Success cases of gene therapy
Hereditary
blindness
Parkinson's
disease
Immune
deficiencies
cancer
Blood disease Hemophilia
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