Current and novel therapeutic strategies for the management of cystic fibrosis

Abstract

ABSTRACT Introduction: Cystic fibrosis (CF), is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene and affects thousands of people throughout the world. Lung disease is the leading cause of death in CF patients. Despite the advances in treatments, the management of CF mainly targets symptoms. Recent CFTR modulators however target common mutations in patients, alleviating symptoms of CF. Unfortunately, there is still no approved treatments for patients with rare mutations to date. Areas covered: This paper reviews current treatments of CF that mitigate symptoms and target genetic defects. The use of gene and drug delivery systems such as viral or non-viral vectors and nano-compounds to enhance CFTR expression and the activity of antimicrobials against chronic pulmonary infections respectively, will also be discussed. Expert opinion: Nano-compounds tackle biological barriers to drug delivery and revitalize antimicrobials, anti-inflammatory drugs and even genes delivery to CF patients. Gene therapy and gene editing are of particular interest because they have the potential to directly target genetic defects. Nanoparticles should be formulated to more specifically target epithelial cells, and biofilms. Finally, the development of more potent gene vectors to increase the duration of gene expression and reduce inflammation is a promising strategy to eventually cure CF.