Problem
Cystic fibrosis is a genetic disease that affects 30,000 Americans, with 1,000 new cases every year. Median age of survival is only 37 years, making it the top killer in genetic disorders.  One of the worse symptoms of the disease is the clogging and inflammation of the lungs due to excess mucus. This problem causes major discomfort and pain to the patient, forcing them to cough up phlegm almost constantly. Cystic fibrosis is a life long plague with no cure.  If this problem could be solved, thousands of people worldwide would benefit. As our mentor stated, “This is an excellent topic to pursue.  An acquaintance of mine had Cystic Fibrosis and his difficulty breathing was always evident.  Wouldn’t it be wonderful if something can be done to alleviate their suffering.” http://www3.nbnet.nb.ca/normap/cfstats.htm

http://www.medicalook.com/diseases_images/cystic_fibrosis.jpg

The cystic fibrosis gene is located on chromosome seven and in cystic fibrosis patients; a DNA mutation in this gene involves the production of a protein called (Cystic Fibrosis Transmembrane conductance Regulator), a part of the cell membrane in certain special epithelial (surface) cells that generate mucus. These special cells might line the airways of the nose and lungs or the stomach wall.  One missing amino acid in the proteins calls for the majority of cystic fibrosis cases. The gene region has 230,000 DNA base pairs that spell out a series of 1,480 amino acids that curl up to make the CFTR protein. The little triangle in the diagram below shows the location of the three-base-pair deletion mutation.

http://www.science.ca/images/scientists/s1-tsui.jp

A normal gene makes CFTR that regulates the passage of chloride ions and hence the secretion of mucus in epithelial (surface) cells lining lungs. This is the corrected gene that we posses in our hands today.  However, the major hindrance that has been experienced so far in administering this gene is an immune system rejection because of the chemical properties of the nanoparticles used to deliver the gene. Our team’s approach was to find a finite tactic to overcome such a hindrance.