New pulmonary therapies directed at targets other than CFTR

Scott H. Donaldson, Luis Galietta

Research output: Contribution to journalArticle

14 Citations (Scopus)

Abstract

Our current understanding of the pathogenesis of cystic fibrosis (CF) lung disease stresses the importance of the physical and chemical properties of the airway surface liquid (ASL). In particular, the loss of cystic fibrosis transmembrane conductance regulator (CFTR) chloride channel function in CF reduces the volume and fluidity of the ASL, thus impairing mucociliary clearance and innate antimicrobial mechanisms. Besides direct approaches to restoring mutant CFTR function, alternative therapeutic strategies may also be considered to correct the basic defect of impaired salt and water transport. Such alternative strategies are focused on the restoration of mucociliary transport by (1) reducing sodium and fluid absorption by inhibiting the ENaC channel; (2) activating alternative chloride channels; and (3) increasing airway surface hydration with osmotic agents. Therapeutic approaches directed at targets other than CFTR are attractive because they are potentially useful to all patients irrespective of their genotype. Clinical trials are underway to test the efficacy of these approaches.

Original languageEnglish
JournalCold Spring Harbor perspectives in medicine
Volume3
Issue number6
DOIs
Publication statusPublished - Jun 2013

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Cystic Fibrosis Transmembrane Conductance Regulator
Mucociliary Clearance
Chloride Channels
Cystic Fibrosis
Lung
Bridge clearances
Pulmonary diseases
Surface Properties
Fluidity
Liquids
Hydration
Chemical properties
Lung Diseases
Restoration
Therapeutics
Physical properties
Salts
Sodium
Genotype
Clinical Trials

ASJC Scopus subject areas

  • Medicine(all)

Cite this

New pulmonary therapies directed at targets other than CFTR. / Donaldson, Scott H.; Galietta, Luis.

In: Cold Spring Harbor perspectives in medicine, Vol. 3, No. 6, 06.2013.

Research output: Contribution to journalArticle

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