notes GENE THERAPY - Gene Therapy for Diabetes Nature This...

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Gene Therapy for Diabetes ( Nature, 11/23/2000 ) This topic reviews and ties together much you’ve learned already in the course. Problem: Type I diabetes - no insulin released by beta cells of pancreas ( cells destroyed by autoimmunity.) - insulin injections are painful, difficult to time with changing glucose levels. Possible solution: Use gene therapy, i.e., insert a gene to make insulin in another cell type (or organ) that can respond to increases in blood glucose. How to get the gene “inserted”? use a virus ( see p.418 ) 1. Get an adenovirus. 2. Add the gene to it to so it can make insulin ( a polypeptide) 3. Inject the new (recombinant, genetically modified) virus into the blood stream so it will infect the liver cells. 4. Liver cells will now incorporate the virus’s DNA, so they can make insulin!! How to get the liver to make insulin at the right time ( after a meal when glucose is high?) - It turns out, the pancreas is not the only glucose-sensing organ.
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