Cystic Fibrosis is a genetic disease that affects the digestive and respiratory system in the body of over 70,000 adults and children worldwide. It is identified by the secretion of thick mucus that blocks the lungs causing deadly infections, and by stopping the pancreatic enzymes from breaking down and absorbing food. Individuals having Cystic Fibrosis suffer from persistent coughing with phlegm, frequent lung infections, poor growth, weight gain and shortness of breath. There are several ways in which this disease can be treated but the most recent attempt is by using gene therapy.
Gene therapy is a treatment used for correcting mutant genes which cause diseases. In most gene therapies, the normal gene is inserted into the cell to replace the defective gene, the gene is transported using modified viruses known as vectors. Gene therapy for Cystic Fibrosis is based on the discovery of the CFTR defective gene (cystic fibrosis transmembrane conductance regulator), by replacing the defective CFTR gene with a functioning CFTR gene. It is a better choice to use gene therapy as a treatment for CF due to its longer lasting results. This discovery was a breakthrough in the way CF was treated.