Cystic FibrosisCystic fibrosis is an incurable hereditary disorder (disease) that mainly affects the respiratory system (airways and the lungs) and the digestive system (stomach, intestines, colon and other organs involved in the digestive process). Cystic fibrosis involves changes in the chemical properties of mucus, causing thicker-than-normal mucus that can lead to obstructions in the respiratory and digestive systems.In the respiratory system, the abnormal mucus obstructs airways and also creates conditions that lead to repeated infections in the lungs, which leads to breathing problems, infections and ultimately, death.In the digestive system, the abnormal mucus can obstruct ducts ...view middle of the document...
One in 40 Caucasian people are healthy carriers of the cystic fibrosis gene. Three decades ago most babies born with cystic fibrosis died in early childhood, but advances in diagnosing and treating the disease have significantly improved the chances of survival.The disease can be managed by:*Antibiotics for lung infections caused by bacteria.*Decongestants, bronchodilators (drugs that open airways congested with mucus) and anti-inflammatory drugs.*Chest or back clapping (to help loosen mucus from lungs) and postural drainage (to help drain mucus from lungs).*Pancreatic enzymes (to help with digestion).*A diet rich in proteins and calories.*Vitamins and other dietary supplements (to add more nutrients to the diet).*Gene TherapyAlthough there is currently no cure for CF, there is a lot of research under way to try to find a cure for CF lung disease through gene therapy. Gene therapy is essentially the removing and replacing of a particular gene or set of genes that are dysfunctional with normal functioning genes in an effort to cure a genetic disorder. Gene therapy is performed by using a vector to insert tiny fragments of replacement DNA into a diseased cell's nucleus. A vector is simply a shuttle that is used to carry DNA into a cell and then leave it there for the cell to use. Generally, the vectors used in gene therapy are viruses because a virus can hold onto the DNA as it enters cells, and then deliver the DNA to the cell. The vector is injected into the defective target cells which scientists hope will absorb the new functional gene, and integrate it into the cells' own genetic makeup. Once this occurs, the inserted gene should function normally, creating the necessary proteins that were deficient before the therapy. After the treatment, the patient should become healthy as their new genes begin to work normally.Scientists have been working for years to find a safe, effective method of gene therapy to treat Cystic Fibrosis. There are many possible vectors that can be used to carry replacement DNA into a cell. Viruses have proven to be the most effective vector for the treatment of Cystic Fibrosis so far but success is still very limited with many trailed viruses having destructive side effects.Scientists have been able to effectively make use of the Adreno-Associated Virus (AAV) to carry replacement genes to defective Cystic Fibrosis cells in the respiratory tract in test subjects. At present, tests with AAV's have been administered in the form of nose drops or drizzling it down a bronchoscope (flexible tube)...
