
Dr. Dietrich Stephan: New Drugs that Repair Broken Genes
Talking Biotech with Dr. Kevin Folta
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Huntington's Disease
Shuntington's disease is among the most horrific diseases that any of us will hopefully never encounter. One copy of a gen called huntington has a tri nucletie repet expansion in xon. And when that happens, the resultant protein has with polyglutomine run in it. So there are more of these glotomine molecules that cause the protein to become very sticky and aggregate within neurons. We believe that's the dominant mechanism behind the disease. Those intra neuronal aggregates actually kill the neurons.
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