Special Issue "Therapeutic Approaches for Cystic Fibrosis"
A special issue of International Journal of Molecular Sciences (ISSN 1422-0067). This special issue belongs to the section "Molecular Pathology, Diagnostics, and Therapeutics".
Deadline for manuscript submissions: closed (31 March 2020).
Special Issues and Collections in MDPI journals
Cystic fibrosis (CF)-causing mutations have complex effects on the CF transmembrane conductance regulator (CFTR) protein, including disrupting its processing to and stability at the plasma membrane, as well as its chloride channel activity. Because of this complex disease etiology, a combination of CFTR modulators is required to rescue the trafficking and functional defects of disease-associated CFTR variants. Despite the success of these combinatorial therapeutics, some CF-causing mutations appear less responsive. For these CF variants, mutation-agnostic therapies have to be developed, such as ENAC inhibitors, activators of alternative chloride channels, and artificial anion transporters (anionophores), as well as cell-based and gene therapies.
This Special Issue on "Cystic Fibrosis" will gather reviews and original articles focused on novel therapeutic approaches to this disease at basic, translational, and clinical levels to provide expert insights and perspectives on advances in the field.
Dr. Nicoletta Pedemonte
Dr. Darren Hutt
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 papers will be 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 100 words) can be sent to the Editorial Office for announcement on this website.
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. International Journal of Molecular Sciences is an international peer-reviewed open access semimonthly journal published by MDPI.
Please visit the Instructions for Authors page before submitting a manuscript. There is an Article Processing Charge (APC) for publication in this open access journal. For details about the APC please see here. 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.
- CFTR channel
- CFTR modulators
- siRNA screening
- alternative chloride channel
- airway epithelium
- gene editing
- cell-based approach