Mechanism of Gene Modification Therapy for Cystic Fibrosis
A special issue of Biomolecules (ISSN 2218-273X). This special issue belongs to the section "Molecular Genetics".
Deadline for manuscript submissions: 28 October 2026 | Viewed by 149
Special Issue Editors
Interests: cystic fibrosis; epithelial transport systems; CF modulators; mRNA therapies
Special Issue Information
Dear Colleagues,
Gene modification therapies offer a targeted approach to correcting the underlying defect in cystic fibrosis (CF), which is caused by mutations in the CFTR gene. mRNA-based methods deliver functional CFTR transcripts to airway epithelial cells, enabling the transient production of the CFTR protein without any changes to the genome. In contrast, gene-editing technologies such as CRISPR/Cas aim to permanently repair pathogenic mutations at the DNA level. Both strategies seek to restore chloride and bicarbonate transport, thereby improving airway hydration and mucociliary clearance. However, effective delivery systems and minimizing immune activation remain key challenges. Advances in precision and safety are essential for achieving long-lasting therapeutic benefits in CF patients.
This Special Issue will highlight recent advances in the molecular mechanisms of gene modification therapies for cystic fibrosis. Topics will include mRNA-based approaches, gene editing technologies, and delivery strategies. We invite the submission of original research articles and reviews that address mechanistic insights, safety considerations, and translational perspectives in CF gene therapy.
Prof. Dr. Wolf-Michael Weber
Dr. Joerg Grosse-Onnebrink
Guest Editors
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 250 words) can be sent to the Editorial Office for assessment.
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. Biomolecules 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 2700 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.
Keywords
- cystic fibrosis
- CFTR
- gene modification therapy
- mRNA therapy
- gene editing
- CRISPR/Cas
- airway epithelium
- ion transport
- nanoparticle delivery
- precision medicine
Benefits of Publishing in a Special Issue
- Ease of navigation: Grouping papers by topic helps scholars navigate broad scope journals more efficiently.
- Greater discoverability: Special Issues support the reach and impact of scientific research. Articles in Special Issues are more discoverable and cited more frequently.
- Expansion of research network: Special Issues facilitate connections among authors, fostering scientific collaborations.
- External promotion: Articles in Special Issues are often promoted through the journal's social media, increasing their visibility.
- Reprint: MDPI Books provides the opportunity to republish successful Special Issues in book format, both online and in print.
Further information on MDPI's Special Issue policies can be found here.
