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Gene Therapy

Gene Therapy. Phung Nguyen. Brief Overview. What Gene Therapy Is The Different Methods of Gene Therapy Ex vivo: indirect injection Types of tissue removed and process Viral and Non-viral In vivo: direct injection Viral and Non-Viral Uses and Costs of Gene Therapy.

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Gene Therapy

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  1. Gene Therapy Phung Nguyen

  2. Brief Overview • What Gene Therapy Is • The Different Methods of Gene Therapy • Ex vivo: indirect injection • Types of tissue removed and process • Viral and Non-viral • In vivo: direct injection • Viral and Non-Viral • Uses and Costs of Gene Therapy

  3. What is Gene Therapy? Purpose: treating genetic disorders with the use of vectors to introduce healthy DNA in a host with damaged DNA.

  4. Viral • Retrovirus • Murine leukemia virus • RNA to DNA • Tends to cause cancer • Lentivirus • Made from more dangerous viruses • Ebola, HVI, and others. • Can effect non-dividing cells • Adenovirus • Such as the common cold virsus • Requires readministration • Adeno-associated virus • Not lethal to human • Can infect dividing and non-diving cells alike

  5. Adeno-associated virus • Has not been found to cause illness in humans. • No immune response from humans • Has been shown to infect chromosome 19 with a 100% success rate with the help of a rep gene. • Makes the infection more predictable and lessen chances of complications • Has a very low uptake frequency • Usually does not combine with host genome • Needs a helper virus to replicate • A lysogenic pathway unlikely without that helper virus

  6. Non-Viral • Naked DNA • DNA simply goes into cell (much like transformation) • Safest method but least efficient • Lipid Carriers • Genetic information shuttled in a liposome • Gene Gun • Genes covered and gold and shot into the body • Electroporation • Shock of electricity to cause pores to form in cell

  7. Lipid Carriers (lipoplex) • Most commonly used are cationic lipids • Have a positive charge to compact negative DNA • Forms complex with DNA • Lipoplex • Promotes Endocytosis • Protects DNA from degradation

  8. Mechanism of Infection • Lipoplex • Enters host cell through Endocytosis • Lipoplex is degraded and DNA is released • DNA is taken up into the nucleus • Recombination occurs and DNA is in genome • Adeno-associated virus • Binds to receptors on cell membrane • Virus particles injected into cell • (process not fully understood) • Gene is expressed by host cell • But usually not taken up into genome

  9. Lipoplex Cationic lipid complex with DNA (Lipoplex)

  10. Adeno-associated virus

  11. Practical Uses and Cost • Has already been implemented in many clinical trials all around the world since 1990. • Has been shown to treat cystic fibrosis, cancer, arthritis, Parkinson’s, and other illnesses caused by genetic mishaps. • Cost is not yet known. • Not readily available to the masses • Mostly still only seen in clinical trails • No Bioshock quite yet • Ethical Cost?

  12. Alternative Methods • Protein Therapy • Simply injects needed protein into patients body • Temporary Solution • Difficult to deliver proteins into cells • Hard to get proteins into cell

  13. The Future of Gene Therapy?

  14. Citations Verma, Inder M. and NikunjSomia. "Gene therapy - promises, problems, and prospects." NATURE 389 (1997): 239-242. Lovejoy, Katherine. "JYI Volume Five Features: Gene Therapy: Techniques of Cell Transfection." Journal of Young Investigators. Feb. 2002. Web. 24 Apr. 2012. <http://www.jyi.org/volumes/volume5/issue5/features/lovejoy.html>. Miller, Dusty A. and Carol Buttimore. "Redesign of Retrovirus Packaging Cell Lines To Avoid." Molecular and Cellular Biology 6.8 (1989): 2895-2902. Clare E. Thomas, AnjaEhrhardt and Mark A. Kay. "PROGRESS AND PROBLEMS WITH THE USE OF VIRAL VECTORS FOR GENE THERAPY." Nature 4 (2003): 346-359. T Niidome and L Huang. "Gene Therapy Progress and Prospects: Nonviral Vectors." Nature.com. Nature Publishing Group, 2002. Web. 24 Apr. 2012. <http://www.nature.com/gt/index.html>. Mueller, C., and T. R. Flotte. "Clinical Gene Therapy Using Recombinant Adeno-associated Virus Vectors." Gene Therapy 15.11 (2008): 858-63. Print. Carter, B. "Adeno-associated Virus Vectors." Current Biology 2.12 (1992): 644. Print. Gonçalves, Manuel AFV. "Adeno-associated virus: from defective virus to effective vector." Virology Journal 2.43 (2005): 1-17. Ghosh, S. "Liver-directed Gene Therapy: Promises, Problems and Prospects at the Turn of the Century." Journal of Hepatology 32 (2000): 238-52. Print. Kotin, R. M. "Site-Specific Integration by Adeno-Associated Virus." Proceedings of the National Academy of Sciences 87.6 (1990): 2211-215. Print. Friedmann, T., and R. Roblin. "Gene Therapy for Human Genetic Disease?" Science 175.4025 (1972): 949-55. Print. Wagner, Darcy E., and Sarit B. Bhaduri. "Progress and Outlook of Inorganic Nanoparticles for Delivery of Nucleic Acid Sequences Related to Orthopedic Pathologies: A Review." Tissue Engineering Part B: Reviews (2011): 110906083848008. Print.

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