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1,320 results for “cystic fibrosis”
Global Functional Genomics Reveals GRK5 as a Therapeutic Target for Cystic Fibrosis
<p>Cystic Fibrosis (CF) is a life-shortening disease affecting >90,000 individuals worldwide predominantly with respiratory symptoms. About 80% of individuals with CF have the F508del mutation that causes the CF transmembrane conductance regulator (CFTR) protein to misfold and be targeted for premature degradation by the endoplasmic reticulum (ER) quality control (ERQC), thus preventing its plasma membrane (PM) traffic. Despite the recent approval of a ‘highly effective’ drug rescuing F508del-CFTR, maximal lung function improvement is ~14% and the drug-targeted genes remain unknown.</p> <p>To identify global modulators of F508del traffic, we performed a high-content siRNA microscopy-based screen of >9,000 genes and monitored F508del-CFTR PM rescue in human airway cells. This primary screen identified 227 F508del-CFTR traffic regulators, of which 35 could be validated by additional siRNAs. Subsequent mechanistic studies established GRK5 as a robust regulator whose inhibition rescues F508del-CFTR PM traffic, thus emerging as a novel potential drug target for CF.</p>
Global burden of non-tuberculous mycobacteria in the cystic fibrosis population: A systematic review and meta-analysis
<p><span><strong>Background</strong>:</span><span> People living with cystic fibrosis have an increased risk of lung infection with non-tuberculous mycobacteria (NTM), which is reportedly increasing. We conducted a systematic review of the literature to estimate the burden (prevalence and incidence) of non-tuberculous mycobacteria in the cystic fibrosis population. </span></p> <p><span><strong>Methods</strong>: Electronic databases, registries, and grey literature sources were searched for cohort and cross-sectional studies reporting epidemiological measures (incidence and prevalence) of NTM infection or NTM pulmonary disease (NTM-PD) in cystic fibrosis. The last search was conducted in September 2021; we included reports since database creation and registry reports published since 2010. The methodological quality of studies was appraised with the Joanna Briggs Institute tool. A random-effects meta-analysis was conducted to summarize the prevalence of NTM infection, and the remaining results are presented in a narrative synthesis. </span></p> <p><span><strong>Results</strong>: Ninety-five studies were included in this review. All 95 studies reported on NTM infection, and 14 of these also reported on NTM-PD. The pooled estimate for the point prevalence of NTM infection was 7.9% (CI 95%, 5.1–12.0%). In meta-regression, sample size and geographical location of the study modified the estimate. Longitudinal analysis of registry reports showed an increasing trend in NTM infection prevalence between 2010 and 2019. </span></p> <p><span><strong>Conclusions</strong>: The overall prevalence of NTM infection in CF is 7.9% and is increasing over time based on international registry reports. Future studies should report screening frequency, microbial identification methods, and incidence rates of progression from NTM infection to pulmonary disease.</span></p>
Whole blood RNA-seq demonstrates an increased host immune response in individuals with cystic fibrosis who develop nontuberculous mycobacterial pulmonary disease
<p><strong>Background </strong></p> <p>Individuals with cystic fibrosis have an elevated lifetime risk of colonization, infection, and disease caused by nontuberculous mycobacteria. A prior study involving non-cystic fibrosis individuals reported a gene expression signature associated with susceptibility to nontuberculous mycobacteria pulmonary disease (NTM-PD). In this study, we determined whether people living with cystic fibrosis who progress to NTM-PD have a gene expression pattern similar to the one seen in the non-cystic fibrosis population. <strong> </strong></p> <p><strong>Methods</strong></p> <p>We evaluated whole blood transcriptomics using bulk RNA-seq in a cohort of cystic fibrosis patients with samples collected closest in timing to the first isolation of nontuberculous mycobacteria. The study population included patients who did (n = 12) and did not (n = 30) develop NTM-PD following the first mycobacterial growth. Progression to NTM-PD was defined by a consensus of two expert clinicians based on reviewing clinical, microbiological, and radiological information. Differential gene expression was determined by DESeq2.</p> <p><strong>Results</strong></p> <p>No differences in demographics or composition of white blood cell populations between groups were identified at baseline. Out of 213 genes associated with NTM-PD in the non-CF population, only two were significantly different in our cystic fibrosis NTM-PD cohort. Gene set enrichment analysis of the differential expression results showed that CF individuals who developed NTM-PD had higher expression levels of genes involved in the interferon (α and γ), tumor necrosis factor, and IL6-STAT3-JAK pathways. <strong> </strong></p> <p><strong>Conclusion</strong></p> <p>In contrast to the non-cystic fibrosis population, the gene expression signature of patients with cystic fibrosis who develop NTM-PD is characterized by increased innate immune responses.</p>
Bright Light Therapy for Depression Symptoms in Adults With Cystic Fibrosis (CF) and COPD
ClinicalTrials.gov study NCT04921332. IPD Sharing: YES. Countries: 1. Publications: 3.
Evaluation of Lung Clearance Index in Cystic Fibrosis (CF) Patients, Infected With P.Aeruginosa
ClinicalTrials.gov study NCT02248922. IPD Sharing: UNDECIDED. Countries: 1. Publications: 1.
Study to Evaluate the Safety & Tolerability of MRT5005 Administered by Nebulization in Adults With Cystic Fibrosis
ClinicalTrials.gov study NCT03375047. IPD Sharing: YES. Countries: 1. Publications: 1.
Dose-finding Study to Assess the Efficacy, Safety and Tolerability of Tobramycin Inhalation Powder in Patients With Non-Cystic Fibrosis Bronchiectasis and Pulmonary P. Aeruginosa Infection
ClinicalTrials.gov study NCT02712983. IPD Sharing: YES. Countries: 6. Publications: 2.
Whole blood RNA-seq demonstrates an increased host immune response in individuals with cystic fibrosis who develop nontuberculous mycobacterial pulmonary disease
Open the record for dataset details and reuse information.
Global burden of non-tuberculous mycobacteria in the cystic fibrosis population: A systematic review and meta-analysis
Open the record for dataset details and reuse information.
Safety and tolerability of a low glycemic load dietary intervention in adults with cystic fibrosis: A pilot study
<p><strong>Context</strong>: To achieve and maintain adequate weight, people with cystic fibrosis (CF) may often consume energy-dense, nutrient-poor foods high in added sugars and refined carbohydrates; however, little is known about the glycemic and metabolic effects of dietary composition in this patient population.</p> <p><strong>Objective</strong>: To investigate the safety and tolerability of a low glycemic load (LGL) diet in adults with CF and abnormal glucose tolerance (AGT).</p> <p><strong>Design</strong>: Prospective, open-label pilot study.</p> <p><strong>Intervention</strong>: Each participant followed his/her typical diet for eight weeks, then transitioned to an LGL diet via meal delivery service for eight weeks.</p> <p><strong>Participants</strong>: Ten adults with CF and AGT completed the trial. Mean age was 27.0±2.1 years, 64% were female, and all had pancreatic insufficiency.</p> <p><strong>Outcome Measures</strong>: The primary outcome was a change in weight from baseline to study completion, with safety established if no significant decline was noted. Other key safety outcomes included change in hypoglycemia measured by patient reports and continuous glucose monitoring (CGM). Exploratory outcomes included changes in other CGM measures, body composition by dual-energy X-ray absorptiometry (DXA), and patient-reported outcomes.</p> <p><strong>Results</strong>:<strong> </strong>There were no significant changes in weight or in subjectively-reported or objectively-measured hypoglycemia. Favorable non-significant trends were noted in CGM measures of hyperglycemia and glycemic variability, DXA measures of fat mass, and gastrointestinal symptom surveys. </p> <p><strong>Conclusions</strong>:<strong> </strong>An LGL dietary intervention was safe and well tolerated in adults with CF and AGT. These results lay the groundwork for future trials investigating the impact of low-glycemic dietary interventions on metabolic outcomes in the CF population.</p>
"Global Functional Genomics Reveals GRK5 as a Therapeutic Target for Cystic Fibrosis", CFTR interactomes
<p><em>A tidy selection of published CFTR interactomes (or CFTR-related omics datasets)</em></p> <p>Collects data from the original online sources, converts gene/protein identifiers into a standard tidy format and updates the identifiers. The Uniprot ID is taken as reference and all other identifiers are genereated from them. This causes some datasets to end up with less genes/proteins than reported.</p> <p>Datasets included</p> <ul> <li><strong>Botelho (2021)</strong> - CFTR traffic regulators <ul> <li>Botelho <em>et al</em> (2022), submitted</li> </ul> </li> <li><strong>Pankow (2015)</strong> - CFTR interactome <ul> <li>Pankow <em>et al</em> (2015) deltaF508 CFTR interactome remodelling promotes rescue of cystic fibrosis. <em>Nature</em>. 528, 510--516. <a href="https://doi.org/10.1038/nature15729">https://doi.org/10.1038/nature15729</a></li> </ul> </li> <li><strong>Canato (2018)</strong> - CFTR interactome <ul> <li>Canato <em>et al</em> (2018) Proteomic interaction profiling reveals KIFC1 as a factor involved in early targeting of F508del-CFTR to degradation. <em>Cell Mol Life Sci</em>. 75(24):4495-4509. <a href="https://doi.org/10.1007/s00018-018-2896-7">https://doi.org/10.1007/s00018-018-2896-7</a></li> </ul> </li> <li><strong>Santos (2019)</strong> - CFTR interactome <ul> <li>Santos <em>et al</em> (2019) Folding Status Is Determinant over Traffic-Competence in Defining CFTR Interactors in the Endoplasmic Reticulum. <em>Cells</em>. 8(4):353. <a href="https://doi.org/10.3390/cells8040353">https://doi.org/10.3390/cells8040353</a></li> </ul> </li> <li><strong>Hutt (2018)</strong> - CFTR interactome <ul> <li>Hutt <em>et al</em> (2018) A Proteomic Variant Approach (ProVarA) for Personalized Medicine of Inherited and Somatic Disease. <em>J Mol Biol</em>. 430: 2951-2973. <a href="https://doi.org/10.1016/j.jmb.2018.06.017">https://doi.org/10.1016/j.jmb.2018.06.017</a></li> </ul> </li> <li><strong>Rauniyar (2014)</strong> - CFTR proteome <ul> <li>Rauniyar <em>et al</em> (2014) Quantitative Proteomic Profiling Reveals Differentially Regulated Proteins in Cystic Fibrosis Cells. <em>J Proteome Res</em>. 13(11): 4668-4675. <a href="https://doi.org/10.1021/pr500370g">https://doi.org/10.1021/pr500370g</a></li> </ul> </li> <li><strong>Almaça (2013)</strong> - ENaC regulome <ul> <li>Almaça <em>et al</em> (2013) High-content siRNA screen reveals global ENaC regulators and potential cystic fibrosis therapy targets. <em>Cell</em>. 154(6):1390-400. <a href="https://doi.org/10.1016/j.cell.2013.08.045">https://doi.org/10.1016/j.cell.2013.08.045</a></li> </ul> </li> <li><strong>Tomati (2018)</strong> - CFTR regulome <ul> <li>Tomati <em>et al</em> (2018) High-throughput screening identifies FAU protein as a regulator of mutant cystic fibrosis transmembrane conductance regulator channel. <em>J Biol Chem</em>. 293(4):1203-1217. <a href="https://doi.org/10.1074/jbc.m117.816595">https://doi.org/10.1074/jbc.m117.816595</a></li> </ul> </li> <li><strong>Simpson (2012)</strong> - Secretome <ul> <li>Simpson <em>et al</em> (2012) Genome-wide RNAi screening identifies human proteins with a regulatory function in the early secretory pathway. <em>Nature Cell Biology</em>. 14, 764-774. <a href="https://doi.org/10.1038/ncb2510">https://doi.org/10.1038/ncb2510</a></li> </ul> </li> <li><strong>Wang (2006)</strong> - CFTR interactome <ul> <li>Wang <em>et al</em> (2006) Hsp90 Cochaperone Aha1 Downregulation Rescues Misfolding of CFTR in Cystic Fibrosis. <em>Cell</em>. 127(4):803-815. <a href="https://doi.org/10.1016/j.cell.2006.09.043">https://doi.org/10.1016/j.cell.2006.09.043</a></li> </ul> </li> <li><strong>Reilly (2017)</strong> - CFTR interactome <ul> <li>Reilly <em>et al</em> (2017) Targeting the PI3K/Akt/mTOR signalling pathway in Cystic Fibrosis. <em>Sci Rep</em>. 9;7(1):7642. <a href="https://doi.org/10.1038/s41598-017-06588-z">https://doi.org/10.1038/s41598-017-06588-z</a></li> </ul> </li> <li><strong>Gilchrist (2006)</strong> - Secretome <ul> <li>Gilchrist <em>et al</em> (2006) Quantitative Proteomics Analysis of the Secretory Pathway. <em>Cell</em>. 127(6):1265-1281. <a href="https://doi.org/10.1016/j.cell.2006.10.036">https://doi.org/10.1016/j.cell.2006.10.036</a></li> </ul> </li> <li><strong>Pankow (2019)</strong> - CFTR interactome <ul> <li>Pankow <em>et al</em> (2019) A posttranslational modification code for CFTR maturation is altered in cystic fibrosis. <em>Science Signaling</em>. 12(562):eaan7984. <a href="https://doi.org/10.1126/scisignal.aan7984">https://doi.org/10.1126/scisignal.aan7984</a></li> </ul> </li> <li><strong>Dang (2020)</strong> - CF lung disease modifier genes <ul> <li>Dang <em>et al</em> (2020) Mining GWAS and eQTL data for CF lung disease modifiers by gene expression imputation. <em>PLoS One</em>. 15(11):e0239189. <a href="https://doi.org/10.1371/journal.pone.0239189">https://doi.org/10.1371/journal.pone.0239189</a></li> </ul> </li> <li><strong>Hodos (2020)</strong> - CF genomic meta-analysis <ul> <li>Hodos <em>et al</em> (2020) Integrative genomic meta-analysis reveals novel molecular insights into cystic fibrosis and deltaF508-CFTR rescue. <em>Sci Rep</em> 10(1):20553. <a href="http://dx.doi.org/10.1038/s41598-020-76347-0">http://dx.doi.org/10.1038/s41598-020-76347-0</a></li> </ul> </li> </ul>
Cystic fibrosis autoantibody signatures associate with Staphylococcus aureus lung infection or cystic fibrosis-related diabetes
<p>While cystic fibrosis (CF) lung disease is characterized by persistent inflammation and infections, and chronic inflammatory diseases are often accompanied by autoimmunity, autooimmune reactivity in CF has not been studied in depth. In this work, we undertook an unbiased approach to explore the systemic autoantibody repertoire in CF. Our results show higher levels of several new autoantibodies in the blood of people with CF (PwCF) compared to control subjects. Some of these are IgA autoantibodies targeting neutrophil components or autoantigens linked to neutrophil-mediated tissue damage in CF. We also found that PwCF with higher systemic IgM autoantibody levels have lower prevalence of <em>S. aureus</em> infection. On the other hand, IgM autoantibody levels in <em>S. aureus</em>-infected PwCF correlate with lung disease severity. Diabetic PwCF have significantly higher levels of IgA autoantibodies in their circulation compared to nondiabetic PwCF, and several of their IgM autoantibodies are associated with worse lung disease. In contrast, in nondiabetic PwCF blood levels of IgA autoantibodies correlate with lung disease. We have also identified other autoantibodies in CF that are associated with <em>P. aeruginosa</em> airway infection. In summary, we have identified several new autoantibodies and associations of autoantibody signatures with specific clinical features in CF.</p>
High prevalence of lipopolysaccharide mutants and R2-Pyocin susceptible variants in Pseudomonas aeruginosa populations sourced from cystic fibrosis lung infections
<p>Chronic, highly antibiotic-resistant infections in cystic fibrosis (CF) lungs contribute to increasing morbidity and mortality. <em>Pseudomonas</em> <em>aeruginosa</em>, a common CF pathogen, exhibits resistance to multiple antibiotics, contributing to antimicrobial resistance (AMR). These bacterial populations display genetic and phenotypic diversity, but it is unclear how this diversity affects susceptibility to bacteriocins. R-pyocins, i.e. bacteriocins produced by <em>P. aeruginosa</em>, are phage-tail-like antimicrobials. R-pyocins have potential as antimicrobials, however, recent research suggests the diversity of <em>P. aeruginosa</em> variants within CF lung infections leads to varying susceptibility to R-pyocins. This variation may be linked to changes in lipopolysaccharide (LPS), acting as the R-pyocin receptor. Currently, it is unknown how frequently R-pyocin-susceptible strains are in chronic CF lung infection, particularly when considering the heterogeneity within these strains. In this study, we tested R2-pyocin susceptibility of 139 <em>P. aeruginosa</em> variants from 17 sputum samples of seven CF patients and analyzed LPS phenotypes. We found that 83% of sputum samples did not have R2-pyocin-resistant variants, while nearly all samples contained susceptible variants. There was no correlation between LPS phenotype and R2-pyocin susceptibility, though we estimate that about 76% of sputum-derived variants lack an O-specific antigen, 40% lack a common antigen, and 24% have altered LPS cores. The absence of a correlation between LPS phenotype and R-pyocin susceptibility suggests LPS packing density may play a significant role in R-pyocin susceptibility among CF variants. Our research supports the potential of R-pyocins as therapeutic agents, as many infectious CF variants are susceptible to R2-pyocins, even within diverse bacterial populations.</p>
Long Term Administration of Inhaled Mannitol in Cystic Fibrosis
ClinicalTrials.gov study NCT00630812. IPD Sharing: Not stated. Countries: 7. Publications: 11.
A Study Evaluating the Long-term Safety and Efficacy of Elexacaftor/Tezacaftor/Ivacaftor in Cystic Fibrosis (CF) Particpants 6 Years and Older and F/MF Genotypes
ClinicalTrials.gov study NCT04545515. IPD Sharing: NO. Countries: 10. Publications: 2.
Safety and Efficacy of Sildenafil in Cystic Fibrosis (CF) Lung Disease
ClinicalTrials.gov study NCT00659529. IPD Sharing: NO. Countries: 1. Publications: 2.
Safety, Tolerability, Pharmacokinetics, and Efficacy of JBT-101 (Lenabasum) in Cystic Fibrosis
ClinicalTrials.gov study NCT02465450. IPD Sharing: Not stated. Countries: 7. Publications: 1.
A Study to Evaluate Efficacy of Ivacaftor in Subjects With Cystic Fibrosis Who Have a 3849 + 10KB C→T or D1152H CFTR Mutation
ClinicalTrials.gov study NCT03068312. IPD Sharing: Not stated. Countries: 1. Publications: 1.
Dose Escalation Study of KB001 in Cystic Fibrosis Patients Infected With Pseudomonas Aeruginosa
ClinicalTrials.gov study NCT00638365. IPD Sharing: Not stated. Countries: 1. Publications: 1.
Study of VX-809 Alone and in Combination With VX-770 in Cystic Fibrosis (CF) Patients Homozygous or Heterozygous for the F508del-CFTR Mutation
ClinicalTrials.gov study NCT01225211. IPD Sharing: Not stated. Countries: 7. Publications: 4.
ScienceDex guides
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These curated guides explain access requirements, typical timelines, costs, and reuse considerations for widely used research datasets.
Allen Brain Atlas
Allen Brain Atlas is an Allen Institute collection of brain map atlases, datasets, APIs, and analysis tools covering mouse, human, and non-human primate brain resources.
Annotated Behaviour and Observability Dataset (ABODe)
ABODe is a University of Edinburgh DataShare dataset for behavior classification in group-housed mice using home-cage video, identities, bounding boxes, ground-plate positions, and annotator labels.
DANDI Archive for NWB datasets
DANDI is a BRAIN Initiative archive for publishing and sharing neurophysiology data, including electrophysiology, optophysiology, and behavioral data packaged as NWB and related standards.
International Brain Laboratory public data
The International Brain Laboratory public data releases expose standardized mouse decision-making experiments, including Neuropixels recordings, widefield calcium imaging, behavior, and session metadata accessed through the ONE API.
OpenNeuro
OpenNeuro is a free, open platform for sharing neuroimaging datasets, with public search, dataset pages, and download paths for web, S3, DataLad, and the OpenNeuro CLI.