Search results for "Cystic Fibrosi"
showing 10 items of 115 documents
Calcifediol-loaded liposomes for local treatment of pulmonary bacterial infections.
2017
The influence of vitamin D3 and its metabolites calcifediol (25(OH)D) and calcitriol on immune regulation and inflammation is well described, and raises the question of potential benefit against bacterial infections. In the current study, 25(OH)D was encapsulated in liposomes to enable aerosolisation, and tested for the ability to prevent pulmonary infection by Pseudomonas aeruginosa. Prepared 25(OH)D-loaded liposomes were nanosized and monodisperse, with a negative surface charge and a 25(OH)D entrapment efficiency of approximately 23%. Jet nebulisation of liposomes was seen to yield an aerosol suitable for tracheo-bronchial deposition. Interestingly, 25(OH)D in either liposomes or ethanol…
Targeting Nonsense: Optimization of 1,2,4-Oxadiazole TRIDs to Rescue CFTR Expression and Functionality in Cystic Fibrosis Cell Model Systems
2020
Cystic fibrosis (CF) patients develop a severe form of the disease when the cystic fibrosis transmembrane conductance regulator (CFTR) gene is affected by nonsense mutations. Nonsense mutations are responsible for the presence of a premature termination codon (PTC) in the mRNA, creating a lack of functional protein. In this context, translational readthrough-inducing drugs (TRIDs) represent a promising approach to correct the basic defect caused by PTCs. By using computational optimization and biological screening, we identified three new small molecules showing high readthrough activity. The activity of these compounds has been verified by evaluating CFTR expression and functionality after…
Strategies against nonsense: oxadiazoles as translational readthrough-inducing drugs (TRIDs)
2019
This review focuses on the use of oxadiazoles as translational readthrough-inducing drugs (TRIDs) to rescue the functional full-length protein expression in mendelian genetic diseases caused by nonsense mutations. These mutations in specific genes generate premature termination codons (PTCs) responsible for the translation of truncated proteins. After a brief introduction on nonsense mutations and their pathological effects, the features of various classes of TRIDs will be described discussing differences or similarities in their mechanisms of action. Strategies to correct the PTCs will be presented, particularly focusing on a new class of Ataluren-like oxadiazole derivatives in comparison …
SOLID LIPID NANOPARTICLES FOR APPLICATIONS IN GENE THERAPY: A REVIEW OF THE STATE OF THE ART
2010
Importance of the field. Gene therapy represents a new paradigm in the prevention and treatment of many inherited and acquired diseases, including genetic disorders, such as cystic fibrosis, haemophilia and many somatic diseases, such as tumours, neurodegenerative diseases and viral infections, such as AIDS. Areas covered in this review. Among a large array of non-viral transfection agents used for in-vitro applications, cationic SLNs are the topic of this review, being recently proposed as an alternative carrier for DNA delivery, due to many technological advantages such as large-scale production from substances generally recognized as safe, good storage stability and possibility of steam …
Oral health of cystic fibrosis patients at a north american center: A pilot study
2019
Background The objective of this study was to describe the oral health status of Cystic Fibrosis (CF) children in a US facility. Material and Methods Twenty CF children ages 6-18 were recruited from Children’s Hospital of Wisconsin Pulmonary Clinic. Parents completed a health questionnaire. Clinical examinations checked dental caries using the dmft/DMFT index, dental hygiene using the Simplified Greene-Vermillion Index (DI-S), gingival inflammation using the Community Periodontal Index of Treatment Needs, and enamel defects using the modified Developmental Defects of Enamel Index. Results The majority (90%) brush twice a day, 65% consume sugary snacks, and 70% visit the dentist every 6 mont…
Prevalence of diabetes mellitus and impaired glucose tolerance in cystic fibrosis.
1990
The aim of this study was to evaluate the prevalence of impaired glucose tolerance or diabetes mellitus in 99 patients (53 M, 46 F; mean age 10.5 +/- 6.9 years), with cystic fibrosis. Glucose tolerance was evaluated in all patients without overt diabetes using the oral glucose tolerance test (OGTT). Six patients showed a pathological OGTT and 2 patients had insulin-requiring diabetes mellitus. The mean age of the patients with impaired glucose tolerance was significantly higher than that of the subjects with normal glucose metabolism (p less than 0.0001). Patients with overt diabetes mellitus were the oldest subjects in the study group.
Impact of hypertonic saline nebulisation combined with oscillatory positive expiratory pressure on sputum expectoration and related symptoms in cysti…
2018
Abstract Objective To evaluate the impact of combining nebulised hyaluronic acid plus hypertonic saline (HA + HS) with oscillatory positive expiratory pressure (oscillatory-PEP) on sputum expectoration and related symptoms in adults with cystic fibrosis (CF). Design Randomised crossover trial. Setting Seven centres. Participants Twenty-two outpatients with CF. Interventions Usual care (HA + HS followed by autogenic drainage) and combined therapy (HA + HS with oscillatory-PEP followed by autogenic drainage]. Each treatment was performed for 5 days. Main outcome measures Sputum expectoration was measured during the nebulisation period (primary outcome), during autogenic drainage and for 24 ho…
Development and Validation of the AdT-Physio Scale: A Tool to Assess Adherence and Perception of Physical Therapist Intervention in Patients With Cys…
2019
Abstract Objective The purpose of this study was to develop a scale to measure patient adherence to physical therapist intervention and to report psychometric properties in patients with cystic fibrosis (CF). Methods This observational, multicenter, qualitative, and cross-sectional study was divided into 2 phases: development of scale items (content validity) and assessment of psychometric properties (construct validity, reliability, and convergent/discriminant validity). Recruited were 121 patients with CF (aged ≥16 years). Sociodemographic characteristics, lung function testing, Coping with Stress Self-Efficacy, brief Coping Orientation to Problems Experienced inventory, and the scale of …
Impact of SARS-CoV-2 infection in patients with cystic fibrosis in Spain: Incidence and results of the national CF-COVID19-Spain survey
2020
CF-COVID19-Spain Registry Group.
Risk factors for cystic fibrosis arthropathy: Data from the German cystic fibrosis registry
2021
Epidemiology and potential risk factors for cystic fibrosis arthropathy (CFA) were studied in a relevant cystic fibrosis (CF) patient cohort.Cohort study of patients included in the German CF registry in 2016-2017. Descriptive analysis, exploratory tests and multivariable logistic regression were used to assess prevalence of CFA and associated potential risk factors for adult patients with/without chronic Pseudomonas aeruginosa infection.6069 CF patients aged from 0 to 78 years were analysed. CFA was observed in 4.9% of the patients. Prevalence was significantly higher in adult patients (8.4%) compared to patients18 years (0.7%; p0.0001). Logistic regression analyses in adult patients (n=33…