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Previous Volume 335:179-188 July 18, 1996 Number 3 Next

	Since this article has no abstract, we have provided an extract of the first 100 words of the full text and any section headings.

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Cystic fibrosis is the most common life-shortening autosomal recessive disorder in the white population, affecting approximately 30,000 persons in the United States.¹ It is caused by mutations in a single gene on the long arm of chromosome 7 that encodes the cystic fibrosis transmembrane conductance regulator (CFTR) (Figure 1).² CFTR has multiple functions involving fluid balance across epithelial cells. It acts as a chloride channel activated by cyclic adenosine monophosphate (cAMP)³ and may stimulate other chloride channels⁴ and inhibit sodium absorption by epithelial sodium channels.⁵

View larger version (28K): [in this window] [in a new window]   Figure 1. Gene, Transcript, and Predicted Structure of the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) . . . [Full Text of this Article]

 
Conventional Pulmonary Therapy for Cystic Fibrosis

Clearance of Airway Secretions

Bronchodilator Therapy

Reduction in the Viscoelasticity of Sputum

Antibiotic Therapy

Exacerbations of Pulmonary Infection

Maintenance Antibiotic Therapy

Quinolone Therapy

Inhaled Antibiotic Therapy

Gene Therapy

Pharmacologic Modulation of Ion Transport

Inhibition of Sodium Absorption

Activation of Alternative Chloride Channels

Antiinflammatory-Drug Therapy

Conclusions

Source Information

From the Department of Pediatrics, University of Washington School of Medicine, Seattle.

Address reprint requests to Dr. Ramsey at the Cystic Fibrosis Research Center, University of Washington School of Medicine, Department of Pediatrics, Box 359301, Seattle, WA 98195.

References

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• Kukavica-Ibrulj, I, Levesque, R C (2008). Animal models of chronic lung infection with Pseudomonas aeruginosa: useful tools for cystic fibrosis studies. Lab Anim 42: 389-412 [Abstract] [Full Text]  
• Tai, A (2008). Renal impairment following aminoglycoside therapy in cystic fibrosis. Thorax 63: 936-936 [Full Text]  
• Khalil, H., Chen, T., Riffon, R., Wang, R., Wang, Z. (2008). Synergy between Polyethylenimine and Different Families of Antibiotics against a Resistant Clinical Isolate of Pseudomonas aeruginosa. Antimicrob. Agents Chemother. 52: 1635-1641 [Abstract] [Full Text]  
• Kremer, T. M., Zwerdling, R. G., Michelson, P. H., O'Sullivan, B. P. (2008). Intensive Care Management of the Patient With Cystic Fibrosis. J Intensive Care Med 23: 159-177 [Abstract]  
• Jarad, N. A., Giles, K. (2008). Risk factors for increased need for intravenous antibiotics for pulmonary exacerbations in adult patients with cystic fibrosis. Chronic Respiratory Disease 5: 29-33 [Abstract]  
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• Lee, C. K. K., Boyle, M. P., Diener-West, M., Brass-Ernst, L., Noschese, M., Zeitlin, P. L. (2007). Levofloxacin Pharmacokinetics in Adult Cystic Fibrosis. Chest 131: 796-802 [Abstract] [Full Text]  
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• Barker, M., Hengst, M., Schmid, J., Buers, H-J., Mittermaier, B., Klemp, D., Koppmann, R. (2006). Volatile organic compounds in the exhaled breath of young patients with cystic fibrosis. Eur Respir J 27: 929-936 [Abstract] [Full Text]  
• Phaff, S. J., Tiddens, H. A. W. M., Verbrugh, H. A., Ott, A. (2006). Macrolide resistance of Staphylococcus aureus and Haemophilus species associated with long-term azithromycin use in cystic fibrosis. J Antimicrob Chemother 57: 741-746 [Abstract] [Full Text]  
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• Bernier, S. P., Sokol, P. A. (2005). Use of Suppression-Subtractive Hybridization To Identify Genes in the Burkholderia cepacia Complex That Are Unique to Burkholderia cenocepacia. J. Bacteriol. 187: 5278-5291 [Abstract] [Full Text]  
• Choi, M. Y., Partridge, A. W., Daniels, C., Du, K., Lukacs, G. L., Deber, C. M. (2005). Destabilization of the Transmembrane Domain Induces Misfolding in a Phenotypic Mutant of Cystic Fibrosis Transmembrane Conductance Regulator. J. Biol. Chem. 280: 4968-4974 [Abstract] [Full Text]  
• Vogne, C., Aires, J. R., Bailly, C., Hocquet, D., Plesiat, P. (2004). Role of the Multidrug Efflux System MexXY in the Emergence of Moderate Resistance to Aminoglycosides among Pseudomonas aeruginosa Isolates from Patients with Cystic Fibrosis. Antimicrob. Agents Chemother. 48: 1676-1680 [Abstract] [Full Text]  
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• Duff, A J A (2002). Psychological consequences of segregation resulting from chronic Burkholderia cepacia infection in adults with CF. Thorax 57: 756-758 [Full Text]  
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• Albelda, S. M., Wiewrodt, R., Zuckerman, J. B. (2000). Gene Therapy for Lung Disease: Hype or Hope?. ANN INTERN MED 132: 649-660 [Abstract] [Full Text]  
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• Saiman, L., Burns, J. L., Whittier, S., Krzewinski, J., Marshall, S. A., Jones, R. N. (1999). Evaluation of Reference Dilution Test Methods for Antimicrobial Susceptibility Testing of Pseudomonas aeruginosa Strains Isolated from Patients with Cystic Fibrosis. J. Clin. Microbiol. 37: 2987-2991 [Abstract] [Full Text]  
• Fung, E. B., Barden, E. M., Wasserman, D., Zemel, B. S., Heinrich, B. T., Scanlin, T. F., Stallings, V. A. (1999). A Six-Month Study of Growth and Energy Expenditure in Children with Cystic Fibrosis Taking a Pulmonary Inhalation Medication (rhDNase). J. Am. Coll. Nutr. 18: 330-338 [Abstract] [Full Text]  
• ELBORN, J S (1999). Treatment of Staphylococcus aureus in cystic fibrosis. Thorax 54: 377-378 [Full Text]  
• Dwyer, M. A., Huang, A. J., Pan, C. Q., Lazarus, R. A. (1999). Expression and Characterization of a DNase I-Fc Fusion Enzyme. J. Biol. Chem. 274: 9738-9743 [Abstract] [Full Text]  
• Ballmann, M., Rabsch, P., von der Hardt, H. (1998). Long term follow up of changes in FEV1 and treatment intensity during Pseudomonas aeruginosa colonisation in patients with cystic fibrosis. Thorax 53: 732-737 [Abstract] [Full Text]  
• Pan, C. Q., Dodge, T. H., Baker, D. L., Prince, W. S., Sinicropi, D. V., Lazarus, R. A. (1998). Improved Potency of Hyperactive and Actin-resistant Human DNase I Variants for Treatment of Cystic Fibrosis and Systemic Lupus Erythematosus. J. Biol. Chem. 273: 18374-18381 [Abstract] [Full Text]  
• Ramsey, B. W. (1998). Pseudomonas aeruginosa Infection as a Complication of Therapy in Pancreatic Fibrosis (Mucoviscidosis), by Sterling D. Garrard, et al, Pediatrics, 1951;8:482. Pediatrics 102: 210-213 [Abstract] [Full Text]  
• Pan, C. Q., Lazarus, R. A. (1998). Hyperactivity of Human DNase I Variants. DEPENDENCE ON THE NUMBER OF POSITIVELY CHARGED RESIDUES AND CONCENTRATION, LENGTH, AND ENVIRONMENT OF DNA. J. Biol. Chem. 273: 11701-11708 [Abstract] [Full Text]  
• WELSH, M. J., RAMSEY, B. W. (1998). Research on Cystic Fibrosis . A Journey from the Heart House. Am. J. Respir. Crit. Care Med. 157: S148-S154 [Full Text]  
• FENG, W., GARRETT, H., SPEERT, D. P., KING, M. (1998). Improved Clearability of Cystic Fibrosis Sputum with Dextran Treatment in vitro. Am. J. Respir. Crit. Care Med. 157: 710-714 [Abstract] [Full Text]  
• Farrell, P. M., Shen, G., Splaingard, M., Colby, C. E., Laxova, A., Kosorok, M. R., Rock, M. J., Mischler, E. H. (1997). Acquisition of Pseudomonas aeruginosa in Children With Cystic Fibrosis. Pediatrics 100: e2-e2 [Abstract] [Full Text]  
• Ackerman, M. J., Clapham, D. E. (1997). Ion Channels -- Basic Science and Clinical Disease. NEJM 336: 1575-1586 [Full Text]