Cystic Fibrosis Gene Therapy - A Cure For This Terrible Disease?
Fibrosis
Cystic fibrosis is a genetic disease that affects certain glands in the body, including those responsible for producing mucus and sweat. It is an inherited disease, meaning that this disease is not contracted from some outside source but is rather the effect of an abnormality in some part of a person's genes. This disorder is caused by the absence of a gene known as the cystic fibrosis trans-membrane conductance regulator.
This gene plays an important role in the creation of sweat, digestive juices, and mucus. CF has been known to affect the lungs, gastrointestinal tract, liver, and pancreas. In the lungs, cystic fibrosis causes overproduction of mucus, resulting in inflammation of the affected tissues. This leads to symptoms such as coughing, weakness, and excessive phlegm production. Eventually, bacteria take refuge in the thick mucus and begin to multiply, avoiding the body's immune system thanks to the protection of the mucus, and lead to secondary infections such as pneumonia.
When it affects the digestive system, cystic fibrosis causes production of thickened secretions from the pancreas, which is responsible for producing important digestive enzymes. These thick secretions block the passage from the pancreas to the digestive tract, and also cause irreversible damage to the pancreas itself. The prevention of digestive enzymes from reaching the digestive tract leads to a lack of absorption of important minerals and nutrients from food, and can result in malnutrition.
Current Treatments for Cystic Fibrosis
Most current treatments for cystic fibrosis deal solely with the management of symptoms of the disease. Most of the treatments commonly used on patients are not cures, and simply help to remove mucus secretions and prevent bacterial infections from taking root in the lungs. Scientists have, however, come up with a new and revolutionary treatment that should be able to provide a lasting cure for this disorder when it is fully developed. This treatment is known as gene therapy.
Gene Therapy & Its Role in Treating Cystic Fibrosis
Since cystic fibrosis results from errors in the genes, scientists seek to correct those errors by replacing the erroneous genes in the affected cells with new, correct genes. This is known as gene therapy. The ideal solution is to be able to insert correct genes into all of the cells affected by the gene for cystic fibrosis. Scientists have discovered that it is extremely difficult to get affected cells to take up the modified genes, however, and thus treatments have thus far proven to be relatively ineffective.
New methods of delivering the corrected genes to the affected cells hold great promise, however, and various methods have already entered testing phases. Once scientists perfect the method of cystic fibrosis gene therapy, patients will then have a proper cure for the disease, as opposed to simply preventing the decline of organ function for as long as possible.
Gene therapy faces serious opposition from some sectors however; as there are people who believe that introducing any form of gene into the human body is unethical. Such protests have yet to have any effect on the progress of research into gene therapy methods, but there is the chance that the scientific community will have to deal with such accusations sometime in the future.
Cystic Fibrosis
Michael Marcus, MD .. www.TheDoctorsVideos.com .. Director Pediatric Allergy and Pulmonary -Maimonides Medical Center .. Fellowship Hospital of Philadelphia .. Castle Connolly Top Doctor
Fibrosis Video Rating: 4 / 5
Orignal From: Cystic Fibrosis
0 Comments