Targeting the Root Cause of Cystic Fibrosis

Author(s): Laura Trescott, Joshua Holcomb, Nicholas Spellmon, Cathy Mcleod, Leala Aljehane, Fei Sun, Chunying Li and Zhe Yang

Volume 16, Issue 9, 2015

Page: [937 - 944] Pages: 8

DOI: 10.2174/1389450115999141030144247

Price: $65


Cystic Fibrosis (CF) is a serious genetic condition caused by CF transmembrane conductance regulator (CFTR) mutation. CF patients have shortened lifespan due to airway obstruction, infection, and end-stage lung failure. However, recent development in CF therapy suggests a brighter future for CF patients. Targeting specific CFTR mutations aims to potentiate the channel gating activity of impaired CFTR and restore protein trafficking to the plasma membrane. Gene therapy introduces correct CFTR gene into the affected airway epithelium leading to the functional expression of CFTR in CF patients. This review will sum up the current status in CF-cause targeting therapy.

Keywords: CFTR, chloride transporter, corrector, cystic fibrosis, gene therapy, potentiator.

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