Novel Therapeutic Strategies for Cystic Fibrosis
A Special Issue of International Journal of Molecular Sciences (ISSN 1422-0067) belonging to the section "Molecular Pathology, Diagnostics, and Therapeutics".
Deadline for manuscript submissions: 31 January 2027 | Viewed by 628
Editors
2. Translational Medicine, Research Institute, The Hospital for Sick Children, University of Toronto, Toronto, ON M5G 1E8, Canada
Interests: airway inflammation in cystic fibrosis (CF); L-arginine/nitric oxide (NO) metabolism; lung and airways diseases
2. Translational Medicine, Research Institute, The Hospital for Sick Children, University of Toronto, Toronto, ON M5G 1E8, Canada
Interests: airway inflammation in cystic fibrosis (CF); allergy and asthma; respiratory viral infections
Special Issue Information
Dear Colleagues,
Highly effective modulator therapy has reshaped cystic fibrosis (CF) care—yet important gaps remain. An estimated ~10-15% of people with CF carry CFTR gene variants not eligible for currently approved modulators, and are intolerant or unresponsive to treatment. Even among responders, chronic airway infection and inflammation may persist, and extrapulmonary complications such as CF-related diabetes and liver disease continue to cause morbidity. At the same time, the modulator era raises practical new questions: How do we quantify residual disease activity? Which biomarkers are fit for purpose? And how do we develop effective novel therapies for those who remain without a modulator option?
We invite you to contribute to this Special Issue on emerging therapeutic strategies that extend, complement, or go beyond CFTR modulator therapy. We welcome original research and reviews across the translational spectrum—from target discovery and preclinical development to clinical evaluation and implementation.
Topics of interest include (but are not limited to):
- Next-generation CFTR rescue strategies (novel modulators, read-through approaches, amplifiers);
- Gene therapy, gene editing, and mRNA-based therapies;
- Anti-inflammatory and immunomodulatory strategies in neutrophil-driven airway disease;
- Novel antimicrobial and anti-biofilm approaches;
- Mucolytics and airway surface liquid-restoring treatments;
- Biomarkers for treatment monitoring, risk stratification, and exacerbation prediction;
- Extracellular vesicles as biomarker sources or therapeutic targets;
- Therapies addressing extrapulmonary manifestations of CF;
- Preclinical models enabling therapeutic development;
- Precision medicine approaches and patient stratification.
We look forward to your contributions.
Prof. Dr. Hartmut Grasemann
Guest Editor
Dr. Dominik Funken
Guest Editor Assistant
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Keywords
- cystic fibrosis
- CFTR modulators
- gene therapy
- airway inflammation
- antimicrobial therapy
- biomarkers
- mucus
- extrapulmonary disease
- clinical trials
- precision medicine
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