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Cool SCIENCE!
Gene therapy
Use a virus to heal you?
• YES! Has potential to treat many diseases, including parkinsons
disease, leukemia, SCID, retinal diseases
How does it work?
• Use the virus to code for protein that is not normally expressed
correctly.
What are the issues?
• Localization, how do we get to the right cells?
• It’s a freaking virus! How do we not get people sick?
RNAi for gene therapy
How does it cure people?
• Silence incorrect genes!
• Act as tumor suppressors
• Cure huntingtons (muscle coordination)
• Cellular degeneration
What do you think?