靶向性纤维化的根本原因

Title:Targeting the Root Cause of Cystic Fibrosis

Volume: 16 Issue: 9

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

Affiliation:

关键词: 囊性纤维化跨膜电导调节因子(CFTR)，校正，囊性纤维化，基因治疗，增效剂

摘要: 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.

Cite this article as:

Laura Trescott, Joshua Holcomb, Nicholas Spellmon, Cathy Mcleod, Leala Aljehane, Fei Sun, Chunying Li and Zhe Yang , Targeting the Root Cause of Cystic Fibrosis, Current Drug Targets 2015; 16 (9) . https://dx.doi.org/10.2174/1389450115999141030144247