1 / 14

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.

josie
Download Presentation

Gene Therapy

An Image/Link below is provided (as is) to download presentation Download Policy: Content on the Website is provided to you AS IS for your information and personal use and may not be sold / licensed / shared on other websites without getting consent from its author. Content is provided to you AS IS for your information and personal use only. Download presentation by click this link. While downloading, if for some reason you are not able to download a presentation, the publisher may have deleted the file from their server. During download, if you can't get a presentation, the file might be deleted by the publisher.

E N D

Presentation Transcript

  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.

More Related