Cystic fibrosis is a genetic disorder responsible for the production of a defective transmembrane protein. In recent years, new protein modulators have been developed. They aim to treat the underlying cause of the disease. The results on the biomarkers of the function of the CFTR protein and on the clinical outcomes are very encouraging. However, there is an individual heterogeneity in the response to modulators within a same genotype. Furthermore, clinical trials focus on the most common mutations in the CFTR gene, in particular DF508. Intestinal organoids, a new model of ex vivo study, could offer a quick approach to increase access to effective treatment for all patients with cystic fibrosis regardless of their CFTR genotype. Organoids could enable personalized treatment of cystic fibrosis.
Download full-text PDF |
Source |
|---|
Publication Analysis
Top Keywords
Similar Publications
Psychometric validation of the Cystic Fibrosis Impact Questionnaire (CF-IQ): A patient-reported outcome assessing impacts of cystic fibrosis.
PLoS One
January 2025
INSERM U1151, Université Paris Cité, Centre de Références Maladies Rares Mucoviscidose et Maladies Apparentées, Hôpital Necker Enfants Malades, Paris, France.
The Cystic Fibrosis (CF) Impact Questionnaire (CF-IQ) was qualitatively developed to assess the impact of CF in the context of treatment advancements and increased longevity. This study reports the CF-IQ validation. In this noninterventional validation study, people with CF completed the 40-item CF-IQ and validating patient-reported outcome measures (PROMs) via electronic diaries at enrollment (baseline) and at the 4-week follow-up.
View Article and Find Full Text PDFAquagenic Wrinkling of the Palms - A Good Early Screening Tool for Cystic Fibrosis (CF) in Malnourished Young Infants in Resource Limited Setting.
Indian J Pediatr
January 2025
Pediatric Respiratory Unit, Department of Pediatrics, Christian Medical College, Vellore, Tamilnadu, 632004, India.
Cystic fibrosis in Pakistan: population harbouring rare variants non-responsive to CFTR modulators and the dilemma of poor health facilities.
Mol Biol Rep
January 2025
Centre for Applied Molecular Biology, University of the Punjab, Lahore, Pakistan.
Background: Pathogenic mutations in the CFTR gene disrupt the normal function of the chloride ion channel CFTR protein, resulting in Cystic Fibrosis (C.F.).
View Article and Find Full Text PDFThe dominant lineage of an emerging pathogen harbours contact-dependent inhibition systems.
Microb Genom
January 2025
Department of Pediatrics, Division of Pulmonary, Asthma, Cystic Fibrosis, and Sleep, Emory University School of Medicine, Atlanta, Georgia, USA.
Bacteria from the complex (Smc) are important multidrug-resistant pathogens that cause a broad range of infections. Smc is genomically diverse and has been classified into 23 lineages. Lineage Sm6 is the most common among sequenced strains, but it is unclear why this lineage has evolved to be dominant.
View Article and Find Full Text PDFClinical benefits and risks of remote patient monitoring: an overview and assessment of methodological rigour of systematic reviews for selected patient groups.
BMC Health Serv Res
January 2025
Department of Planning and Development, Region Västra Götaland, NU Hospital Group, Trollhättan, Sweden.
Background: Remote patient monitoring implies continuous follow-up of health-related parameters of patients outside healthcare facilities. Patients share health-related data with their healthcare unit and obtain feedback (which may be automatically generated if data are within a predefined range). The goals of remote patient monitoring are improvements for patients and reduced healthcare costs.
View Article and Find Full Text PDFWant AI Summaries of new PubMed Abstracts delivered to your In-box?
Enter search terms and have AI summaries delivered each week - change queries or unsubscribe any time!