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Purification of the Cystic Fibrosis Transmembrane Conductance Regulator Protein Expressed in Saccharomyces cerevisiae
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CFTR function and prospects for therapy

John R Riordan1

  • 1Department of Biochemistry and Biophysics, Cystic Fibrosis Treatment and Research Center, School of Medicine, University of North Carolina at Chapel Hill, NC 27599, USA. jack_riordan@med.unc.edu

Annual Review of Biochemistry
|February 29, 2008
PubMed

Insights

Cystic fibrosis (CF) is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. This review explores therapeutic strategies based on CFTR

Area of Science:

  • Biochemistry
  • Molecular Biology
  • Physiology

Background:

  • Cystic fibrosis (CF) is a genetic disorder caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
  • CFTR is an epithelial anion channel essential for ion and fluid homeostasis.
  • It belongs to the adenine nucleotide-binding cassette (ABC) transporter family.

Purpose of the Study:

  • To review therapeutic strategies for CF.
  • To discuss CFTR structure, function, and processing.
  • To explore insights from CFTR's biosynthetic pathway, trafficking, and turnover.

Main Methods:

  • Literature review of CFTR structure and function.
  • Analysis of CFTR's role in ion and fluid transport.
  • Examination of CFTR's biosynthetic processing, trafficking, and turnover.

Main Results:

  • Knowledge of CFTR structure and function informs therapeutic development.
  • Understanding CFTR's cellular journey is crucial for effective treatment.
  • Various therapeutic strategies are emerging based on these insights.

Conclusions:

  • Targeting CFTR offers promising therapeutic avenues for cystic fibrosis.
  • Further research into CFTR's complex biology will drive innovation.
  • Comprehensive understanding of CFTR is key to managing CF.

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