A5073987241- Cystic Fibrosis Research Advances
- Neonatal Respiratory Health Research
- Tracheal and airway disorders
- Asthma and respiratory diseases
- RNA Interference and Gene Delivery
- Inhalation and Respiratory Drug Delivery
- Pediatric health and respiratory diseases
- Virus-based gene therapy research
- Nematode management and characterization studies
- Respiratory viral infections research
- Immunodeficiency and Autoimmune Disorders
- Allergic Rhinitis and Sensitization
- Pharmaceutical studies and practices
- Viral gastroenteritis research and epidemiology
- Ion Transport and Channel Regulation
- Congenital Ear and Nasal Anomalies
- Respiratory and Cough-Related Research
- Esophageal and GI Pathology
- Advanced biosensing and bioanalysis techniques
- Infant Nutrition and Health
- Glycosylation and Glycoproteins Research
- Respiratory Support and Mechanisms
- Genomics and Rare Diseases
- Long-Term Effects of COVID-19
- Sarcoidosis and Beryllium Toxicity Research
Université Paris Cité
2016-2025
Assistance Publique – Hôpitaux de Paris
2016-2025
Sorbonne Paris Cité
2013-2025
Inserm
1992-2024
Hôpital Cochin
2013-2024
Institut Cochin
2019-2024
Cystic Fibrosis Foundation
2024
Hôpital Européen Georges-Pompidou
2022
Délégation Paris 5
2012-2021
University of North Carolina Health Care
2020
Elexacaftor–tezacaftor–ivacaftor is a small-molecule cystic fibrosis transmembrane conductance regulator (CFTR) modulator regimen shown to be efficacious in patients with at least one Phe508del allele, which indicates that this combination can modulate single allele. In whose other CFTR allele contains gating or residual function mutation already effectively treated previous modulators (ivacaftor tezacaftor–ivacaftor), the potential for additional benefit from restoring protein unclear.
This is the second in a series of four papers updating European Cystic Fibrosis Society (ECFS) standards for care people with CF. paper focuses on establishing and maintaining health. The guidance produced using an evidence-based framework wide stakeholder engagement, including from CF community. Authors provided narrative description their topic statements, which were more directive. These statements reviewed by Delphi exercise, achieving good levels agreement group all statements....
Rationale: Although in patients with diffuse bronchiectasis (DB) and a normal sweat test the presence of one mutation cystic fibrosis transmembrane conductance regulator (CFTR) gene is frequently observed, its pathogenic role development DB remains unclear.Objectives: To evaluate association between CFTR heterozygosity protein dysfunction airways DB.Methods: Nasal potential difference was measured 122 unknown origin (Cl− < 60 mmol/L). They were classified according to mutations: zero (85...
Our purpose was to characterize the tachykinin receptor type involved in nasal obstruction exogenous substance P rhinitic patients. We also attempted assess biochemical and cellular events associated with this response. Nasal challenges were performed seven patients allergic rhinitis. They received increasing doses (10 80 nmol) of P, neurokinin A, N-terminal fragment P(1-9), saline on 4 different days separated by 14 days. airway resistance (NAR) increased a dose-dependent manner P. Maximal...
Background Although polycations are among the most efficient nonviral vectors for gene transfer, expression they allow is still too low in vivo applications. To engineer more potent polycationic vectors, factors governing intracellular trafficking of a plasmid complexed with current need to be identified. Methods and results The DNA glycosylated polylysines or polyethylenimine (PEI) derivatives was studied by electron microscopy human airway epithelial cells. cellular processing complexes...
Abstract Background To investigate the nuclear import mechanism of plasmid/polyethylenimine (PEI) derivative complexes and putative targeting therapeutic genes by use oligosaccharides, we have studied plasmid DNA complexed either with PEI or lactosylated (Lac–PEI) in cystic fibrosis human airway epithelial cells (∑CFTE29o‐ cells). Methods results Cells were synchronized a double‐thymidine block protocol gene transfer efficiency was evaluated: Lac–PEI‐ PEI‐mediated greatly increased when...
The diagnosis of cystic fibrosis (CF) is based on a characteristic clinical picture in association with sweat chloride (Cl(-)) concentration greater than 60 mmol/L or the identification two CF-causing mutations. A challenging problem significant number children for whom no definitive possible because they present symptoms suggestive CF, level intermediate range between 30 and mmol/L, only one identified mutation.To investigate function transmembrane conductance regulator (CFTR) protein...
BackgroundThe 6 min walk test (6MWT) provides prognostic information in various respiratory diseases, but limited data exist cystic fibrosis (CF) adults.MethodsConsecutive CF adults who performed 6MWT at Cochin Hospital (Paris, France) over 12 years were analyzed. The cut-off walking distance (6MWD) value that best predicted a combined endpoint (death without transplant or lung transplant) was established using receiver operating curve. Determinants of low 6MWD desaturation (SpO2 ≤ 90%)...
<h2>Abstract</h2> Elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) was shown to be safe and efficacious in people with cystic fibrosis (CF) heterozygous for <i>F508del</i> a minimal function mutation (<i>F</i>/MF) or homozygous (<i>F</i>/<i>F</i>) two pivotal Phase 3 trials, significantly improving percentage predicted forced expiratory volume 1 second, Cystic Fibrosis Questionnaire–Revised, Respiratory Domain (CFQ-R RD) scores, sweat chloride concentration. Here, we analyzed the 11...
We aimed to characterise lymphoid neogenesis in bronchiectasis and cystic fibrosis (CF) lungs examine the role of bacterial infection. Lymphoid aggregates were examined using immunohistochemical staining morphometric analysis surgical lung sections obtained from nonsmokers patients with or CF. Sterile, Pseudomonas aeruginosa - Staphylococcus aureus -coated agarose beads instilled intratracheally mice. Kinetics chemokine expression over 14 days. scarce human nonsmokers, but numerous...
Progressive cystic fibrosis (CF) lung disease is the main cause of mortality in CF patients. starts early childhood. With current standards care, respiratory function remains largely normal children and more sensitive outcome measures are needed to monitor disease. Chest CT currently most imaging modality pulmonary structural changes adolescents with CF. To quantify reliably among multiple centres, standardisation chest protocols needed. SCIFI (Standardised Imaging Framework for...