Recent advances and new perspectives in targeting CFTR for therapy of cystic fibrosis and enterotoxin-induced secretory diarrheas

Weiqiang Zhang, Naoaki Fujii, Anjaparavanda P. Naren

Research output: Contribution to journalReview article

19 Citations (Scopus)

Abstract

The cystic fibrosis transmembrane conductance regulator (CFTR) is a cAMP-regulated chloride channel localized primarily at the apical surfaces of epithelial cells lining airway, gut and exocrine glands, where it is responsible for transepithelial salt and water transport. Several human diseases are associated with an altered channel function of CFTR. Cystic fibrosis (CF) is caused by the loss or dysfunction of CFTR-channel activity resulting from the mutations on the gene; whereas enterotoxin-induced secretory diarrheas are caused by the hyperactivation of CFTR channel function. CFTR is a validated target for drug development to treat these diseases. Significant progress has been made in developing CFTR modulator therapy by means of high-throughput screening followed by hit-to-lead optimization. Several oral administrated investigational drugs are currently being evaluated in clinical trials for CF. Also importantly, new ideas and methodologies are emerging. Targeting CFTR-containing macromolecular complexes is one such novel approach.

Original languageEnglish (US)
Pages (from-to)329-345
Number of pages17
JournalFuture Medicinal Chemistry
Volume4
Issue number3
DOIs
StatePublished - Mar 1 2012

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Cystic Fibrosis Transmembrane Conductance Regulator
Enterotoxins
Cystic Fibrosis
Diarrhea
Therapeutics
Exocrine Glands
Investigational Drugs
Macromolecular Substances
Chloride Channels
Salts
Epithelial Cells
Clinical Trials
Mutation
Water
Pharmaceutical Preparations
Genes

All Science Journal Classification (ASJC) codes

  • Drug Discovery
  • Pharmacology
  • Molecular Medicine

Cite this

Recent advances and new perspectives in targeting CFTR for therapy of cystic fibrosis and enterotoxin-induced secretory diarrheas. / Zhang, Weiqiang; Fujii, Naoaki; Naren, Anjaparavanda P.

In: Future Medicinal Chemistry, Vol. 4, No. 3, 01.03.2012, p. 329-345.

Research output: Contribution to journalReview article

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