Novel Therapeutic Approaches for Cystic Fibrosis

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

Deadline for manuscript submissions: 20 May 2024 | Viewed by 1036

Special Issue Editors


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Guest Editor
Department of Molecular Medicine, University of Padova, 35131 Padova, Italy
Interests: biochemistry and computational chemistry; cystic fibrosis; ferroptosis; lipid peroxidation; neurodegeneration; cancer reasearch; protein phoshorylation; signal transduction; drug design and development; drug repurposing
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Guest Editor
Department of Agrifood, Environmental and Animal Sciences, University of Udine, Vis Cotonificio 108, 33100 Udine, Italy
Interests: protein kinases; protein phosphorylation; kinase; signal transduction; apoptosis; cell signaling; signaling; cancer cell signaling; cellular biochemistry; cancer research; cystic fibrosis; nanotechnologies
Special Issues, Collections and Topics in MDPI journals

E-Mail Website
Guest Editor
Department of Molecular Medicine, University of Padova, 35131 Padova, Italy
Interests: cystic fibrosis; ferroptosis; lipid peroxidation; neurodegeneration; analitical biochemistry and cellular biology

Special Issue Information

Dear Colleagues,

Mutations in the gene-encoding CFTR (Cystic Fibrosis Transmembrane Conductance Regulator), a chloride and bicarbonate channel located on the apical membrane of epithelial cells, trigger Cystic Fibrosis (CF), a severe, rare disease. The primary cause of death from CF is respiratory failure, driven by infections, prolonged inflammation, and damage to the lungs. The complex and varied clinical symptoms affecting several organs, including the pancreas, liver, kidneys, and intestines, further emphasize the significant contribution of this single protein to the pathogenesis of CF. Around 2000 mutations in the CFTR protein have been discovered, classified into six categories. Most CF patients bear the F508del class II mutation, which causes CFTR entrapment in the endoplasmic reticulum and its premature degradation before reaching the plasma membrane. Even though many drugs have been approved and clinical care ensures a steady improvement in the quality of life of CF patients, the development of new therapies not limited to the most common mutations but also targeting other pathological CFTR mutants remains a challenge. With this Special Issue, entitled "Novel Therapeutic Approaches for Cystic Fibrosis", we would like to invite authors to contribute review articles or original research articles that shed light on recent advances in the discovery of new therapeutic perspectives for CF. Contributions can include, but are not limited to, new therapies targeting orphan CFTR mutations, innovative treatments that could control CF symptoms or prevent CF complications, and studies elucidating the mechanisms of action of promising new molecules or suggesting alternative approaches and pharmaceutical strategies in the management of CF.

Dr. Giorgio Cozza
Dr. Andrea Venerando
Dr. Ilaria Artusi
Guest Editors

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Keywords

  • cystic fibrosis
  • CFTR mutations
  • pathophysiology
  • inflammation
  • infection
  • high-throughput screening
  • drug development
  • therapies
  • treatments
  • clinical trials

Published Papers (1 paper)

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9 pages, 1520 KiB  
Opinion
Unity Is Strength: The Mutual Alliance between CFTR and SLC26A6 as Therapeutic Opportunity in Cystic Fibrosis
by Marilena Pariano, Cinzia Antognelli, Luigina Romani and Claudio Costantini
Pharmaceuticals 2024, 17(3), 367; https://doi.org/10.3390/ph17030367 - 12 Mar 2024
Viewed by 710
Abstract
Patients with cystic fibrosis (PwCF) have recently experienced an unprecedented breakthrough with the adoption of modulator therapy in clinical practice. This remarkable achievement has led to the reconsideration of disease management as the increased life expectancy has gradually shifted the attention over a [...] Read more.
Patients with cystic fibrosis (PwCF) have recently experienced an unprecedented breakthrough with the adoption of modulator therapy in clinical practice. This remarkable achievement has led to the reconsideration of disease management as the increased life expectancy has gradually shifted the attention over a spectrum of extra-pulmonary manifestations that become prevalent in the aging population. It comes to be that complementary approaches that target patient co-morbidities are needed for the optimal clinical management of PwCF. A strategy would be to adjuvate the cystic fibrosis transmembrane conductance regulator (CFTR) in performing its functions in the different organs in which it is expressed. Solute carrier family 26 (SLC26) members appear ideal in this context. Indeed, they not only cooperate with CFTR in the organ-dependent regulation of ion fluxes but physically interact with it to reciprocally modulate their function. In this opinion, we summarize available evidence pointing to a physical and functional interaction between CFTR and SLC26 members, with a particular focus on SLC26A6 for its wider expression and broader anion selectivity, and then discuss how restoring the physical interaction between CFTR and SLC26A6 might be beneficial in the treatment of PwCF in the era of modulator therapy. Full article
(This article belongs to the Special Issue Novel Therapeutic Approaches for Cystic Fibrosis)
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