The University of Nebraska Medical Center has been selected as one of 12 sites to participate in a study using gene therapy to treat cystic fibrosis.
The genetic disease affects approximately 30,000 children and adults in the United States.
With cystic fibrosis, a defective gene causes the body to produce an abnormally thick, sticky mucus that clots the lungs and leads to chronic, life-threatening lung infections.
The infections are responsible for shortening the life expectancy for CF patients to about 33 years.
Gene therapy is an experimental procedure being developed to treat a variety of diseases by putting normal copies of genes into cells that need them.
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What is Cystic Fibrosis?
Cystic fibrosis (CF) is a chronic, progressive, and frequently fatal genetic disease of the body's mucus glands. CF primarily affects the respiratory and digestive systems in children and young adults.
The sweat glands and the reproductive system are also usually involved. On the average, individuals with CF have a lifespan of approximately 30 years.
CF-like disease has been known for over two centuries. The name, cystic fibrosis of the pancreas, was first applied to the disease in 1938.
How common is CF?
According to the data collected by the Cystic Fibrosis Foundation, there are about 30,000 Americans, 3,000 Canadians, and 20,000 Europeans with CF.
The disease occurs mostly in whites whose ancestors came from northern Europe, although it affects all races and ethnic groups.
Accordingly, it is less common in African Americans, Native Americans, and Asian Americans. Approximately 2,500 babies are born with CF each year in the United States.
Also, about one every 20 Americans is an unaffected carrier of an abnormal "CF gene." These 12 million people are usually unaware that they are carriers.
What are the symptoms of CF?
CF does not follow the same pattern in all patients but affects different people in different ways and to varying degrees.
However, the basic problem is the same-an abnormality in the glands, which produce or secrete sweat and mucus. Sweat cools the body; mucus lubricates the respiratory, digestive, and reproductive systems, and prevents tissues from drying out, protecting them from infection.
People with CF lose excessive amounts of salt when they sweat. This can upset the balance of minerals in the blood, which may cause abnormal heart rhythms. Going into shock is also a risk.
Mucus in CF patients is very thick and accumulates in the intestines and lungs.
The result is malnutrition, poor growth, frequent respiratory infections, breathing difficulties, and eventually permanent lung damage. Lung disease is the usual cause of death in most patients.
CF can cause various other medical problems. These include sinusitis (inflammation of the nasal sinuses, which are cavities in the skull behind, above, and on both sides of the nose), nasal polyps (fleshy growths inside the nose), clubbing (rounding and enlargement of fingers and toes), pneumothorax (rupture of lung tissue and trapping of air between the lung and the chest wall), hemoptysis (coughing of blood), cor pulmonale (enlargement of the right side of the heart), abdominal pain and discomfort, gassiness (too much gas in the intestine), and rectal prolapse (protrusion of the rectum through the anus).
Liver disease, diabetes, inflammation of the pancreas, and gallstones also occur in some people with CF.
Source: http://www.nhlbi.nih.gov/health/public/lung/other/cf.htm (National Institutes of Health).