1. Gene Therapy

2. Genetic Engineering of Bacteria to Produce Human Proteins
human cell
Human insulin gene
bacterial DNA
Insert bacterial/human DNA into bacterial cell
Bacteria produces human INSULIN protein, which is purified and given to patients

3. Genetic Engineering of Bacteria to Produce Human Proteins
Human blood clotting gene
human cell
bacterial DNA
Insert bacterial/human DNA into bacterial cell
Bacteria produces BLOOD CLOTTING PROTEIN,
which is purified and given to patients

4. Genetic Engineering of Bacteria to Produce Human Proteins
Human erythropoietin gene
human cell
bacterial DNA
Insert bacterial/human DNA into bacterial cell
Bacteria produces ERYTHROPOIETIN PROTEIN,
which is purified and given to patients

5. Gene Therapy
An experimental treatment which attempts to treat a disease by inserting a gene into the cells of a human
>3000 patients have been treated in >300 clinical trials around the globe
No gene therapy treatment is yet approved for general use

6. - received gene therapy for OTC Deficiency
Jesse Gelsinger
- received gene therapy for OTC Deficiency
- died September 1999

7. Gene Therapy for OTC Deficiency
Normal OTC gene
human cell
Insert the OTC gene into a virus
Deliver the virus directly into vein leading to liver  virus infects liver cells and delivers gene  OTC protein is produced inside the person

8. ~ Patricia Simons, December 1999
“I ask you to let these trials continue. It’s the only hope the parents and children have. Don’t make this one more dream differed for them. Sadly, patients die in the name of medical science, and we all know that. Gene therapy is just beginning. Please give it a chance.”
~ Patricia Simons, December 1999

9. Severe Combined Immunodeficiency (SCID)
The first disease to be treated with gene therapy
Persons with SCID lack a functional immune system and are susceptible to severe infections

10. David Vetter ( )
- lived in a sterile “plastic bubble” because had a single defective gene that caused Severe Combined Immunodeficiency (SCID)

11. Gene Therapy for SCID Remove blood cells from patient
Re-infuse the blood cells into the patient
Infect blood cells with a virus that has been genetically engineered to carry the corrected SCID gene

12. Ashanthi DeSilva - inherited SCID -received 1st ever gene therapy in 1990

13. Gene Therapy for SCID Using Long-lived Umbilical Cord Blood Cells
Remove blood cells from the umbilical cord
Re-infuse the cord blood cells into the patient
Infect cord blood cells with a virus that has been genetically engineered to carry the corrected SCID gene

14. Andrew Gobea (& mom) - inherited SCID - received 1st ever gene therapy using long-lived cord blood cells

15. Other Partially Successful Gene Therapy Trials
therapy to induce new blood vessel growth in patients with blocked heart arteries or with other circulatory problems
therapy to induce brain tumor cells to “commit suicide”

16. Ethical Questions Concerning Gene Therapy
Should treatments be used to change the appearance, intelligence, or athletic ability of healthy people?
Should parents be allowed to genetically manipulate their children before birth?
Should gene therapy be allowed that alters germ-line cells (reproductive cells that will pass the altered genes to future generations)?

17. David Vetter ( ) - lived in a sterile “plastic bubble” because had a single defective gene that caused Severe Combined Immunodeficiency (SCID)

18. Ashanthi DeSilva - inherited SCID -received 1st ever gene therapy in 1990

19. Andrew Gobea (& mom) - inherited SCID - received 1st ever gene therapy using long-lived cord blood cells

20. - received gene therapy for OTC Deficiency
Jesse Gelsinger
- received gene therapy for OTC Deficiency
- died September 1999