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Clinical management of CF » CFTR modulators » CFTR modulators: mechanism of action

CFTR modulators: mechanism of action

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CFTR modulators are small molecules that target specific defects caused by mutations in the CFTR gene. They are classified into three main groups: Potentiators, Correctors and Production correctors (sometimes referred to as Read-through agents). This nomenclature describes their mechanism of action, which is determined by the type of mutation they target.1

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References
  1. Sloane PA. Curr Opin Pulm Med 2010;16:591–7
  2. O’Sullivan BP & Freedman SD. Lancet 2009;373:1891–1904
  3. Rogan MP. Chest 2011;139;1480–90
  4. Welsh MJ et al. Cystic fibrosis. In Valle D et al (Eds). OMMBID. The McGraw-Hill Companies Inc 2004. Part 21, chapter 201
  5. Clinicaltrials.gov. http://clinicaltrials.gov/ct2/show/NCT01225211
  6. Adapted from Rogan MP et al. Chest 2011;139;1480–90