Cystic fibrosis (CF) is a group of conditions variable in severity and mainly affecting the upper respiratory system. It is the most common fatal inherited disease and can be traced to a single recessive gene. Improved therapy has transformed CF from a disease characterized by death in early childhood to a chronic illness, with most patients living to adulthood. But despite this progress, there still is no cure for the disease and most patients eventually succumb to infections of the airways and lung failure.
The defective cystic fibrosis gene is carried by 1 in every 20 Caucasians, on average. These carriers do not have symptoms of CF. The condition is only expressed if a person inherits a copy from both parents, a risk for every 1 in 2,000 to 3,000 live births, depending on the country, making CF one of the most common lethal genetic diseases in Caucasians. The incidence is lower in other racial groups.
Cystic fibrosis affects approximately 30,000 children and adults in the USA. 1,000 children with CF are born every year in the USA, of which one quarter will not survive beyond adolescence. According to the U.S. CF Foundation, one half of all individuals with CF die before the age of 31.