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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?
