Therapeutic strategies to correct malfunction of CFTR

M. Lim, P. L. Zeitlin

Research output: Contribution to journalArticlepeer-review

12 Scopus citations


Cystic fibrosis (CF) is a systemic autosomal recessive inherited disorder that results from mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Although the gene was cloned 11 years ago, there still is no definitive treatment to correct the functional deficit. Current treatment strategies focus on pancreatic enzyme replacement and control of pulmonary inflammation and infection. This review examines novel strategies still in preclinical development or phase 1 clinical trials. Gene therapy is an evolving area of study that offers the potential for a cure for cystic fibrosis. CF lung disease is a significant barrier to effective gene delivery and transfer, but new vectors show promise in overcoming these limitations. There are also new pharmacological therapies aimed at correcting defects in CFTR processing and function. These are tailored to the specific class of mutation but may offer therapeutic benefit to many patients. They include phenylbutyrate, flavonoids, aminoglycosides and xanthines.

Original languageEnglish (US)
Pages (from-to)159-164
Number of pages6
JournalPaediatric Respiratory Reviews
Issue number2
StatePublished - 2001
Externally publishedYes


  • Butyrates/pharmacology
  • Chloride channels/metabolism/physiology
  • Cystic fibrosis transmembrane conductance regulator/genetics/physiology
  • Cystic fibrosis/drug therapy/genetics
  • Flavones/therapeutic use
  • Gene therapy/methods
  • Human
  • Molecular chaperones/physiology
  • Mutation/physiology
  • Post-translational/drug effects
  • Protein folding
  • Protein processing

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health
  • Pulmonary and Respiratory Medicine


Dive into the research topics of 'Therapeutic strategies to correct malfunction of CFTR'. Together they form a unique fingerprint.

Cite this