Closer to a Cure for Cystic Fibrosis Essays -- Proteasomes Cystic Fibr

Overcoming Proteasomes: One Step Closer to a Cure for Cystic Fibrosis Take a deep breath and consider how easy it was to do so. Now picture struggling and gasping for air everyday; normal, easy tasks to the average person prove to be quite challenging to a patient of cystic fibrosis. Scientists have always been daunted by this fatal genetic disease that affects the body by excreting a â€Å"thick mucus† in the lungs, making breathing difficult and blocking the â€Å"ducts† leading from the pancreas, causing â€Å"poor digestion of food† (1). Until recently, there was nothing that scientists could do to provide a long-term cure, but even though scientists cannot cure patients one hundred percent, they can increase their life expectancy. Despite having discovered the cystic fibrosis gene’s location in 1989, numerous underlying obstacles prevent complete success (2). The primary obstacle that prevents scientists from making gene therapy an effective cure is the placement of the healthy genes into long-term cells, the cells that remain long enough to be replicated. The replacement of the healthy gene into long-term cells is necessary because these cells make the new cells, thereby distributing the healthy DNA throughout the body. The properly functioning gene is attached to a vector or â€Å"carrying molecule† that will transport â€Å"the therapeutic gene to the patient’s target cells† (2). Currently, the most common vector is a virus because it can easily capture the gene and deliver it into the cell; the virus infiltrates the corrupt cells and places the healthy gene into the nucleus which then transforms the corrupt cell into one which operates properly (2). The only difficulty is that the body’s natural immune system provides many b... ...t-education/tips/ccysfibr.html>. 2. Institute NHGR. Gene Therapy [Internet]. 2007 [2007 September 18]; Available from . 3. Stefano Ferrari DMG, Eric WFW Alton. Barriers to and new approaches for gene therapy and gene delivery in cystic fibrosis. Advanced Drug Delivery Reviews 2002;64: 1373-1393. 4. J Kim C-PC, KG Rice. The proteasome metabolizes peptide-mediated nonviral gene delivery systems. Gene Therapy 2006; 12: 1681-1690. 5. Neil Campbell, and Lawrence Mitchell (1999). Biology. New York, Addison Wesley Longman, Inc. 6. Institute NHGR. Learning About Cystic Fibrosis [Internet]. 2007 [2007 September 18]; Available from: . 7. U Griesenbach DG, and EWFW alton. Gene therapy progress and prospects: cystic fibrosis. Gene Therapy 2007; 13: 1071-1077.