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 “thick mucus” in the lungs, making breathing difficult and
blocking the “ducts” leading from the pancreas, causing “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
“carrying molecule” that will transport “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...
... middle of paper ...
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
6. Institute NHGR. Learning About Cystic Fibrosis [Internet]. 2007 [2007 September 18];
7. U Griesenbach DG, and EWFW alton. Gene therapy progress and prospects: cystic
fibrosis. Gene Therapy 2007; 13: 1071-1077.
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