1. GENE THERAPY -“molecular bandage” -use of DNA as a pharmaceutical, to treat disease

2. STEPS 1.Identify needed gene 2.Isolate a healthy version of the gene 3.Put into recipient - gene will produce needed protein

4. Simple, right? To get the gene in: http://learn.genetics.utah.edu/content/tech/genetherapy/gttools/toolbox.s wf

5. Viral Vectors (in vivo) General advantages of viral vectors: – very good at targeting and entering cells. – might be engineered to target specific types of cells. – can be modified so that they can't replicate and destroy the cell.

6. General drawbacks of viral vectors: – A virus can't "expand" to fit a piece of genetic material larger than it is naturally built to carry, some genes may be too big to fit into a certain type of virus. – Viruses can cause immune responses in patients, resulting in two potential outcomes: Patients may get sick. A patient's immunity to a virus may prevent him from responding to repeated treatments.

7. Types of Viral Vectors retrovirus: gene incorporates – can cause cancer adenovirus – doesn’t integrate: short-lived and immunogenic adeno-associated virus – integrates at a specific site herpes virus – immunogenic, but does not integrate

8. Ex-vivo: isolate patients’ cells treat the cells outside the body liposomes/plasmids – good for ex-vivo: no limit on length

9. SUCCESSFUL USES -retinal disease Leber's congenital amaurosis, -X-linked SCID - ADA-SCID - adrenoleukodystrophy - chronic lymphocytic leukemia (CLL) - acute lymphocytic leukemia (ALL) - multiple myeloma - haemophilia - Parkinson's disease

10. DRAWBACKS... In 2012, Glybera became the first gene therapy treatment to be approved for clinical use in either Europe or the United States after its endorsement by the European Commission (1.6 MILLION DOLLARS PER TREATMENT!!!!!)

11. Slight issues... -EXPENSIVE! -can cause cancer-causing mutations -can change gametes -needs to be delivered to millions of the RIGHT cells -gene doping?