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Gene Therapy: Hemophilia and Beyond

Gene Therapy: Hemophilia and Beyond. By: Joel Rodriguez. What is Hemophilia. The royal disease Bleeding disorder Factors VIII and IX Current treatments – Pros and Cons Clean proteins. Image from: http ://static.ddmcdn.com/gif/blood-cells.jpg.

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Gene Therapy: Hemophilia and Beyond

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  1. Gene Therapy: Hemophilia and Beyond By: Joel Rodriguez

  2. What is Hemophilia • The royal disease • Bleeding disorder • Factors VIII and IX • Current treatments – Pros and Cons • Clean proteins Image from: http://static.ddmcdn.com/gif/blood-cells.jpg

  3. Images from: http://www.daviddarling.info/encyclopedia/H/hemophilia.html

  4. Symptoms • Symptoms may arise when damage is already great. • Profuse bleeding • Bruising • Joint swelling (Can be very extreme) • Simple wounds take longer than normal to stop bleeding

  5. Gene Therapy? • Different techniques • In vivo • Ex vivo • Target organs • Liver • Bone marrow • Stem cells • Muscular tissue • Introduce Viral DNA? Image from: http://www.cosmosmagazine.com/node/1024

  6. Gene Therapy? • Bad track record • Deaths in previous trials • Many problems with gene therapy • Immune system • Inhibitors • Cancer • Permanency?

  7. NY Times Article • British trial showing success • Patients blood factors sustainability improvements • Intravenous injections • Liver is focus • Cost • Standard treatment: upwards of $20 million in a lifetime • Gene therapy (if successful): Approx. $30,000 per injection

  8. NY Times Article • Makes use of adeno-associated virus • Less chance of DNA alteration • Some patients show great response • Less than the article would have you believe • Still not a permanent solution thus far • All comes back to the immune system

  9. Image from: http://upload.wikimedia.org/wikipedia/commons/3/3d/Gene_therapy.jpg

  10. Successes with Hemophilia • Recent successes with new techniques • Lab tests with mice and even dogs give hope • Most improvement with different types of immunosuppressant • Viral vector alone still has major effects. http://pavlovsape.files.wordpress.com/2010/06/lab-rat.jpg

  11. Liu, L., H. Liu, C. Mah, and B. S. Fletcher. "Indoleamine 2,3-dioxygenase Attenuates Inhibitor Development in Gene-therapy-treated Hemophilia A Mice." Gene Therapy 16.6 (2009): 724-33. Print.

  12. Possible applications • Potential use for many other diseases • HIV • Cancer • Diabetes • Not all techniques would work • Ones used now are only temporary

  13. Specific Example • Muscular dystrophy • Another disease that has high hopes with gene therapy • Currently using adeno-associated viruses (same as for hemophilia) • Study also tested different types of AAVs • Quite astonishing result (although temporary)

  14. Wang, Zhong, Tong Zhu, ChunpingQiao, Liqiao Zhou, Bing Wang, Jian Zhang, Chunlian Chen, Juan Li, and Xiao Xiao. "Adeno-associated Virus Serotype 8 Efficiently Delivers Genes to Muscle and Heart." Nature Biotechnology 23.3 (2005): 321-28. Print.

  15. Conclusion • Articles claim of breakthrough is way out of proportion • Still many problems to be tackled • Cell longevity and division reduces effectiveness • The more expensive ex vivo technique holds the key to longevity of cure • Very much worth pursuing for the betterment of mankind as a whole • Imagine a world free of disease

  16. Thanks for listening!Any questions may be asked now.

  17. Works Cited • Chuah, Marinee K. L., Desire Collen, and Thierry VandenDriessche. "Gene Therapy for Hemophilia." The Journal of Gene Medicine 3.1 (2001): 3-20. Print. • Liu, L., H. Liu, C. Mah, and B. S. Fletcher. "Indoleamine 2,3-dioxygenase Attenuates Inhibitor Development in Gene-therapy-treated Hemophilia A Mice." Gene Therapy 16.6 (2009): 724-33. Print. • Wang, Zhong, Tong Zhu, ChunpingQiao, Liqiao Zhou, Bing Wang, Jian Zhang, Chunlian Chen, Juan Li, and Xiao Xiao. "Adeno-associated Virus Serotype 8 Efficiently Delivers Genes to Muscle and Heart." Nature Biotechnology 23.3 (2005): 321-28. Print. • http://www.nytimes.com/2011/12/11/health/research/hemophilia-b-gene-therapy-breakthrough.html?_r=2&src=tp

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