In Caucasians, a specific alteration of the CFTR protein accounts for about 75 percent of the mutant alleles. Regardless of ethnicity, nine in ten people with CF die of respiratory complications. A recent study of over 1000 patients reported in the literature correlated the progression of respiratory disease, as measured by pulmonary function, with the nature of patients' mutations. The rate of disease progression occurred in two phases. The first phase was more gradual than the second and was determined largely by the genotype.
Cystic fibrosis is the most common lethal hereditary disorder affecting children and young adults.
Tremendous progress was made during the past 30 years in increasing life expectancy by aggressively and prophylactically treating the characterisitc pulmonary insufficiency and infections. However, while little progress has been made over the past decade, much hope persists for some form of gene-based therapy. This study shows that, to be effective, any new advances based on gene therapy need to focus on the early course of the disease. The interactivity between the CFTR gene and cystic fibrosis would be limited to the initial phase of the disease. All material on this website is protected by copyright.