Emerging Therapies for the Treatment of Cystic Fibrosis

Dear Colleagues,

Cystic fibrosis (CF) is a common life-shortening rare disease caused by mutations in the gene coding for CF transmembrane conductance regulator (CFTR), a chloride and bicarbonate channel located at the apical membranes of epithelial cells. CF mainly affects lungs, leading to infections, inflammation, and respiratory failure, being the principal cause of mortality. Besides, the complex and heterogeneous clinical manifestations that involve several organs (pancreas, liver, kidneys, and intestine, among others) highlight the crucial role of this single protein in CF pathophysiology. Although the vast majority of CF patients bear the class II F508del mutation, which is retained in the endoplasmic reticulum and degraded before reaching the plasma membrane, over 2000 mutations have been identified in the CFTR protein, grouped into six categories. At present, although several drugs have been approved, and clinical care ensures steady gains in the quality of life of CF patients, the development of new therapies targeting not only the most common mutations but also the orphan ones remains challenging.

With this Special Issue entitled “Emerging Therapies for the Treatment of Cystic Fibrosis”, we would like to invite authors to contribute with review or original research articles dedicated to the most recent advances on the discovery of new perspective therapeutics for CF. Contributions will include but are not limited to emerging therapies targeting different CFTR class mutations, novel treatments that could help to control the symptoms or prevent complications, studies concerning the elucidation of the mechanisms of action of promising CF treating molecules, and those which discuss different approaches and pharmaceutical strategies in this pathology. 

Dr. Giorgio Cozza
Dr. Andrea Venerando
Dr. Valeria Raia
Prof. Dr. Mauro Piacentini
Guest Editors

Manuscript Submission Information

Manuscripts should be submitted online at www.mdpi.com by registering and logging in to this website. Once you are registered, click here to go to the submission form. Manuscripts can be submitted until the deadline. All submissions that pass pre-check are peer-reviewed. Accepted papers will be published continuously in the journal (as soon as accepted) and will be listed together on the special issue website. Research articles, review articles as well as short communications are invited. For planned papers, a title and short abstract (about 100 words) can be sent to the Editorial Office for announcement on this website.

Submitted manuscripts should not have been published previously, nor be under consideration for publication elsewhere (except conference proceedings papers). All manuscripts are thoroughly refereed through a single-blind peer-review process. A guide for authors and other relevant information for submission of manuscripts is available on the Instructions for Authors page. Pharmaceuticals is an international peer-reviewed open access monthly journal published by MDPI.

Please visit the Instructions for Authors page before submitting a manuscript. The Article Processing Charge (APC) for publication in this open access journal is 2900 CHF (Swiss Francs). Submitted papers should be well formatted and use good English. Authors may use MDPI's English editing service prior to publication or during author revisions.