1. Charlie McLaughlin

2. What are Genetic Disorders?  Genetic disorders are illnesses stemming from errors in a person’s genes  Any mistake in a gene can alter how a specific protein is produced  Without proper proteins, the body will not function properly and will take on a chronic and possibly life threatening condition

3. How is it Caused?  Genetic disorders can be congenital meaning they occur from birth or they may develop over time  Congenital disorders occur when parents who are carriers of the disorder pass on their genes to their children  Progressive disorders that take time to set in may be caused by a mutation in DNA

5. What is Gene Therapy?  Gene therapy is the process of taking DNA coded for specific genes and adding it to a person’s genome in efforts to treat and alleviate genetic disorders  There are two types of treatment, In Vivo and Ex Vivo  Regardless of the type, each therapy requires a vector to transport DNA into cells

6. In Vivo Vs. Ex Vivo

7. Viral Vectors  Viral vectors are viruses that have had recombinant DNA inserted into their genome  They are injected into patients and infect cells allowing the new therapeutic gene to be copied  The addition of the “fixed” gene will begin to treat the genetic disorder

9. Non-Viral Vectors  Non-viral vectors are somewhat safer alternatives to viral vectors because they come without an infection risk  They involve different methods of delivery into cells Gold particles laced with DNA Electroporation: electrifying cell membranes to alter permeability Microbubbles and Ultrasounds

10. Non Viral Vectors (cont.)

11. Limitations  Still considered in trial phase  Viral Vectors can be infectious  Patient specific  Under developed technonlogy

12. Future  Higher success rates in patients  Transition to Non viral vectors Hydrodynamic Gene Transfer Jet Gun  Ability to cure and not just treat disorders  Ability to cure more diseases

13. References  Edwards, Mark T. Gene Therapy: Current Treatment Options and Likely Near-Term Developments. Student Pulse: International Student Journal. Vol. 6 NO. 9, 2014.  Wikipedia: Gene Therapy.  Wikipedia: Genetic Disorders.  Ginn, Samantha L., Alexander, Ian E., Edelstein, Michael L., Abedi, Mohammad, R., Wixon, Jo. Gene therapy clinical trials worldwide to 2012 – an update. The Journal of Gene Medicine. Vol 15, Iss 2, p 65– 77, February 2013.Vol 15, Iss 2,