Special Issue "Cystic Fibrosis and Rare Mutations: New Promising Approaches via Proteostase Modulators"

A special issue of Pharmaceuticals (ISSN 1424-8247). This special issue belongs to the section "Biopharmaceuticals".

Deadline for manuscript submissions: closed (15 September 2023) | Viewed by 159

Special Issue Editor

Department of Pharmacy, University of Genoa, Viale Benedetto XV, 16132 Genoa, Italy
Interests: medicinal chemistry; drug design; GPCR; enzyme; neuroprotective agents, cystic fibrosis; QSAR; virtual screening; homology modeling
Special Issues, Collections and Topics in MDPI journals

Special Issue Information

Dear Colleagues,

Cystic fibrosis (CF) is caused by mutations in the CF transmembrane conductance regulator (CFTR) gene, which encodes a cAMP-regulated chloride (Cl-) and bicarbonate (HCO3-) channel expressed at the apical membrane of epithelial cells. The most common mutation—F508del—is present in 80% of individuals with CF worldwide.

New curative treatments aimed at rescuing CFTR dysfunctionality have emerged, although the proven efficacy of some of these treatments has been explored only for particular mutations. A total of 15% of patients cannot be treated with CFTR-directed therapeutics. Hence, there is an interest in finding an alternative strategy to treat patients with CF independently of CFTR mutations.  Pharmacological chaperones (PCs) are correctors that directly bind to the F508del-CFTR mutant and promote its folding and trafficking. Conversely, proteostasis modulators (PMs) act indirectly by altering protein synthesis, trafficking and degradation. The development of new compounds that target the biological components of the CFTR physiological pathway may be useful to optimize combination therapies for those patients that do not respond to the current treatments. In particular, the identification of new candidate drugs acting as PMs for the correction of the basic defect in cystic fibrosis is expected to be of high relevance.

This Special Issue invites both reviews and original articles that shed light on the rational design and development of new PMs to be exploited in CF patients, including virtual screening applications and high-throughput screening (HTS) campaigns. Biological assays that lead to the discovery of novel hit compounds that can be further probed for different CFTR mutants are also included in the scope of the Special Issue. This Special Issue aims to summarize the state of the-art, and the latest findings published in the cystic fibrosis field, as well as to elucidate future directions.

Dr. Elena Cichero
Guest Editor

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.

Published Papers

There is no accepted submissions to this special issue at this moment.
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