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Cystic fibrosis (CF) is the number-one genetic killer of children and young adults in this country. CF causes the body to produce a thick, sticky mucus which clogs the lungs. This abnormal mucus leads to chronic lung infections, fatal lung damage, and also interferes with digestion.
The symptoms of CF are diverse, vary in severity, and are often confused with pneumonia or asthma. A sweat test diagnoses CF by measuring the amount of salt in sweat. An abnormally high salt level indicates that a person has CF.
Approximately 30,000 people in the United States have CF. A child must inherit two defective CF genes -- one from each parent -- to be born with CF. Ten million Americans (one in 29) are symptomless carriers of the defective CF gene. Every day, approximately three babies are born with CF in this country and every day at least one person with CF will die.
In 1955, the Cystic Fibrosis Foundation was founded to benefit, fight and cure cystic fibrosis. This Foundation is one of the most efficient voluntary health organizations of its kind. More than 90 percent of the dollars raised by the Foundation is available for investment in CF research, care and medical programs. Currently, the Foundation supports landmark human gene therapy studies which are evaluating several gene delivery strategies in people with CF.
Despite its magnitude, gene therapy only constitutes one avenue of research funded by the Foundation. Pharmacological approaches study ways to control CF by attacking the chronic infections, and thinning the mucus that ravages the lungs of individuals with CF. The results of these studies will determine the best treatments to alleviate CF-related problems and improve the quality of life for people with CF.
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