Call for papers: Pharmacological approaches to cystic fibrosis
• 大类 : 医学 - 2区
• 小类 : 药物化学 - 1区
Cystic fibrosis (CF) is an autosomal recessive genetic disease caused by mutations in the gene encoding for the cystic fibrosis transmembrane conductance regulator (CFTR), an epithelial anion channel that is involved in the regulation of salt and water absorption and secretion. CFTR is expressed in a variety of cells types, and particularly in epithelial cells of various organs including lungs, pancreas, liver, and intestine. Mutations in the CFTR gene leading to loss of function of the corresponding protein result in a severe multi-organ disease. In the lungs, lack of CFTR-dependent anion secretion leads to abnormally thick and sticky mucus that clogs the small airways impairing mucociliary clearance. Consequently, the lungs become more vulnerable to persistent bacterial infections, which trigger a severe inflammatory response and a progressive loss of respiratory function. In the gastrointestinal tract, pancreatic insufficiency is typically found in CF patients, resulting in malabsorption and failure to thrive. There is a series of therapeutic strategies already used or under development. In particular, rescue of mutant protein is feasible with drugs (correctors, potentiators, amplifiers) that target the specific molecular defects caused by the different types of mutations. In case of undruggable mutations, alternative epithelial proteins are possible as targets. However, targeting the basic defect is not enough, particularly for patients that are at an advanced stage of the disease. In fact, treatment of inflammation and eradication of infection by bacteria highly resistant to present antibiotics are important needs. Furthermore, effective agents to remove the thick mucus clogging the airways of the patients are also needed.
Potential topics for manuscripts include:
Modulators of alternative epithelial ion transport systems