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Previous Volume 352:1992-2001 May 12, 2005 Number 19 Next

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Advances in the care of patients with cystic fibrosis have improved survival, and as a result, patients with the disease now often live beyond the third decade.¹ In addition, developments in the understanding of the genetics and molecular mechanisms of cystic fibrosis have led to new targets for treatment and an increasingly hopeful outlook. This review summarizes the mechanisms underlying the disease, the sequelae stemming from the absence of a functioning cystic fibrosis transmembrane conductance regulator (CFTR), and the therapeutic strategies devised to correct these abnormalities. Progress in the supportive care of patients with the disease has been reviewed elsewhere.^2,3,4

Historical Background

. . . [Full Text of this Article]

Structure and Function of CFTR

Pathobiologic Features of Organ System Disease

Mechanism Underlying the Sweat-Gland Abnormality

Bioelectric Measurements in the Lung

Glandular Secretion and Small-Airway Physiology

Mutations in CFTR And Their Consequences

Interventions Tailored to Specific CFTR Defects

Summary

Source Information

From the Gregory Fleming James Cystic Fibrosis Research Center (S.M.R., S.M., E.J.S.), and the Departments of Medicine (S.M.R., E.J.S.), Pediatrics (S.M.R.), Genetics (E.J.S.), and Physiology and Biophysics (E.J.S.), University of Alabama at Birmingham, Birmingham.

Dr. Rowe and Ms. Miller contributed equally to this article.

Address reprint requests to Dr. Sorscher at the Gregory Fleming James Cystic Fibrosis Research Center, University of Alabama at Birmingham, 1530 3rd Ave. S., MCLM 796, Birmingham, AL 35294, or at sorscher@uab.edu.

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