Treating and Managing Cystic Fibrosis: Patients Clinical Considerations
A special issue of Journal of Personalized Medicine (ISSN 2075-4426). This special issue belongs to the section "Clinical Medicine, Cell, and Organism Physiology".
Deadline for manuscript submissions: closed (10 April 2024) | Viewed by 1645
Special Issue Editors
Interests: CFTR; gene networks; human model systems; translational medicine; biomarker-driven clinical research; drug development; intracellular trafficking; rare mutations
Interests: cystic fibrosis; CFTR; chronic infections; lung disease; GI tract; translational medicine; CFTRD; pancreatitis; CFTR modulators; rare mutations; cell models
Special Issue Information
Dear Colleagues,
Cystic fibrosis (CF) is the most common life-limiting autosomal recessive disease worldwide characterized by clinical manifestations in multiple organ systems. A long-term goal is to understand how global and local properties of complex macromolecular networks influence the mutated cystic fibrosis transmembrane conductance regulator (CFTR) biogenesis, and how changes in such properties can lead to co- and multimorbidities in people with CF. The advent of modulator therapies has stressed the importance of the personalized functional characterization of CFTR genotypes because it is virtually impossible to perform randomized clinical trials to identify the best therapeutics for all CF patients. The cooperation between scientists and clinicians has led to the translation of basic science to biomarker-driven clinical research and shortly into clinical practice.
Given the heterogeneity of clinical presentations of CF and therapeutic responses to modulators, human model systems are essential in management and treatment decisions becoming drivers in personalized medicine.
“Bench to bedside” has the potential to improve patient care, as well as to determine eligibility for future biomarker-driven clinical trials critical for patients with unmet clinical needs. The challenge now is to compare the performance of different human model systems and their capacity to predict clinical outcome measures.
For this Special Issue, we invite review, articles, and original studies addressing the latest developments in human model systems and in predictive biomarkers to improve patient care, providing expert insights and perspectives on advances in the field.
Dr. Fabiana Ciciriello
Dr. Vincenzina Lucidi
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 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. Journal of Personalized Medicine 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 2600 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
- human model systems
- biomarker-driven clinical research
- theratyping
- patient-derived cell models
- CFTR rare mutations
- personalized medicine
- bench to bedside
