Neurobiology of Disease


Date: April 12, 2010 (Monday)
Time: 10:30 a.m.
Theme: Neurodegeneration (Alzheimer's, Parkinson's Disease and Aging)
Module: ToolBox
Session: Gene therapy: cells and viruses
Instructor: Jodie McBride
Description: This session will provide an overview of approaches to gene therapy, focusing on the use of viral-mediated gene transfer.
Study questions:
  1. What factors dictate which gene therapy strategy (viral vectors, stem cells, gene guns, etc.) is best suited for a particular application?
  2. What dictates the tropism of viral vectors and how does this affect its application and potency in the different regions of the brain/body?
  3. What are the benefits and limitations of viral gene therapy to the CNS?
  4. What are the obstacles to overcome when translating gene therapy from the bench top to the clinic?
  5. How does the immune system react to the presence of foreign stem cells, viral vectors used in gene therapy applications?
  6. What are the ethical/moral concerns of using gene therapy to treat disease in humans?
  1. Kay MA, Glorioso JC, Naldini L (2001) Viral vectors for gene therapy: the art of turning infectious agents into vehicles of therapeutics. Nat Med 7:33-40.
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  2. McBride JL, Ramaswamy S, et al (2006) Viral delivery of glial cell line-derived neurotrophic factor improves behavior and protects striatal neurons in a mouse model of Huntington's disease. PNAS 103:9345-9350.
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  3. Müller FJ, Snyder EY, Loring JF (2006) Gene therapy: can neural stem cells deliver? Nat Rev Neurosci 7:75-84.
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