Emerging drug treatments for cystic fibrosis

Pamela L. Zeitlin

Research output: Contribution to journalReview articlepeer-review

9 Scopus citations


Cystic fibrosis (CF) is one of the most common life-shortening inherited disorders. Mutations in the cystic fibrosis transmembrane regulator (CFTR) gene disrupt the localisation and function of the cAMP-mediated chloride channel. Most of the morbidity and mortality arise from the lung disease which is characterised by excessive inflammation and chronic infection. Research into the mechanisms of wild-type and mutant CFTR biogenesis suggest that multiple drug targets can be identified. This review explores the current understanding of the nature of the different mutant CFTR forms and the potential for repair of the chloride channel defect. High-throughput screening, pharmacogenomics and proteomics bring recent technological advances to the field.

Original languageEnglish (US)
Pages (from-to)523-535
Number of pages13
JournalExpert Opinion on Emerging Drugs
Issue number2
StatePublished - Nov 2003
Externally publishedYes


  • Antibiotics
  • Antimicrobial peptide
  • Butyrates
  • Chaperones
  • Chloride
  • Gene therapy
  • Genetic
  • Genomics
  • High-throughput screening
  • Inflammation
  • Isoflavones
  • Mucolytic
  • Mutation
  • Proteomics
  • Vaccine

ASJC Scopus subject areas

  • Pharmacology
  • Pharmacology (medical)


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