Gene Therapy?
Thursday, September 2nd, 2010 at
3:32 pm
Pamela G asked:
how gene therapy might be used to treat cystic fibrosis
Tagged with: Cystic Fibrosis • Gene Therapy
Filed under: Cystic Fibrosis
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Cystic fibrosis is a genetic disorder where the genes in the lungs tell the cells to produce excess phlegm, causing difficulty breathing. Gene therapy in theory would add the correct gene to the phlegm producing cells making the produce the correct amount.
They didn’t teach us this “phlegm theory” in medical school, but cystic fibrosis is typically caused by a genetic defect in a single gene, CFTR, an ion transporter. Without good copies of this gene, cells in the GI tract and lungs, among others, cannot move ions correctly, and this results in mucus deposits in the lungs and pancreatic insufficiency, among other things. In theory, if you could fix this gene in all the relevant cells, you could cure the disease. This is the goal of gene therapy — to provide the gene to these cells. However, there are a lot of barriers and technical problems to delivering genes to intact cells and interest in the field is not as high as it once was.