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 March 2025 | Viewed by 2810
Special Issue Editors
Interests: biochemistry and computational chemistry; cystic fibrosis; ferroptosis; lipid peroxidation; neurodegeneration; cancer reasearch; protein phoshorylation; signal transduction; drug design and development; drug repurposing
Special Issues, Collections and Topics in MDPI journals
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
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
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