Gene Therapy for CF

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New research on gene replacement therapy offers the hope of a cure for cystic fibrosis (CF). This fatal disease, symptomized by coughing, wheezing, lung infections, very salty sweat, and excessive appetite with inability to gain weight, affects about 30,000 Americans.

CF manifests when a person receives a mutated copy of the CFTR gene from both parents. More than 10 million people carry one copy of the defective gene. Most CF patients die in childhood, but some live into their 30s or 40s. Advances in drug therapies and other treatments such as lung transplants have improved the quality of life of CF patients in recent years, but still do not offer a cure.

Because …