Special Issue "Cystic Fibrosis and Rare Mutations: New Promising Approaches via Proteostase Modulators"
Deadline for manuscript submissions: closed (15 September 2023) | Viewed by 159
Interests: medicinal chemistry; drug design; GPCR; enzyme; neuroprotective agents, cystic fibrosis; QSAR; virtual screening; homology modeling
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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
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