Search Results - "Fajac, Isabelle"

Therapeutic Approaches for Patients with Cystic Fibrosis Not Eligible for Current CFTR Modulators by Fajac, Isabelle, Sermet, Isabelle

“…Cystic fibrosis is a severe autosomal recessive disorder caused by mutations in the cystic fibrosis transmembrane conductance regulator ( ) gene encoding the…”
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New drugs, new challenges in cystic fibrosis care by Fajac, Isabelle, Burgel, Pierre-Régis, Martin, Clémence

“…Cystic fibrosis (CF) is a genetic disease caused by variants in the gene encoding for the CF transmembrane conductance regulator (CFTR) protein, a chloride and…”
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Bacteria-driven peribronchial lymphoid neogenesis in bronchiectasis and cystic fibrosis by Frija-Masson, Justine, Martin, Clémence, Regard, Lucile, Lothe, Marie-Noëlle, Touqui, Lhousseine, Durand, Aurélie, Lucas, Bruno, Damotte, Diane, Alifano, Marco, Fajac, Isabelle, Burgel, Pierre-Régis

“…We aimed to characterise lymphoid neogenesis in bronchiectasis and cystic fibrosis (CF) lungs and to examine the role of bacterial infection.Lymphoid…”
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New treatments targeting the basic defects in cystic fibrosis by Fajac, Isabelle, Wainwright, Claire E.

“…Cystic fibrosis (CF) is a monogenic autosomal recessive disorder affecting around 75,000 individuals worldwide. It is a multi-system disease but the main…”
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An innovative phase II trial to establish proof of efficacy and optimal dose of a new inhaled epithelial sodium channel inhibitor BI 1265162 in adults and adolescents with cystic fibrosis: BALANCE-CFTM 1 by Goss, Christopher H, Jain, Raksha, Seibold, Wolfgang, Picard, Anne-Caroline, Hsu, Ming-Chi, Gupta, Abhya, Fajac, Isabelle

“…Inhibition of the epithelial sodium channel (ENaC) represents an important, mutation-agnostic therapeutic approach to restore airway surface liquid in patients…”
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Could a defective epithelial sodium channel lead to bronchiectasis by Fajac, Isabelle, Viel, Marion, Sublemontier, Sébastien, Hubert, Dominique, Bienvenu, Thierry

“…Bronchiectasis is defined as a permanent dilation of the airways arising from chronic bronchial inflammation/infection. In 50% of cases, no etiology can be…”
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Transcription of plasmid DNA: Influence of plasmid DNA/polyethylenimine complex formation by Honoré, Isabelle, Grosse, Stéphanie, Frison, Natacha, Favatier, Florence, Monsigny, Michel, Fajac, Isabelle

“…Polyethylenimine (PEI) is one of the most potent non-viral vectors. We have developed a lactosylated PEI (Lac-PEI) to enhance cell-specific transfection and…”
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Potocytosis and cellular exit of complexes as cellular pathways for gene delivery by polycations by Grosse, Stéphanie, Aron, Yolande, Thévenot, Guiti, François, Dominique, Monsigny, Michel, Fajac, Isabelle

“…Background Although polycations are among the most efficient nonviral vectors for gene transfer, the gene expression they allow is still too low for in vivo…”
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New horizons for cystic fibrosis treatment by Fajac, Isabelle, De Boeck, Kris

“…Cystic fibrosis is an inherited multi-system disease associated with chronic lung infection, malabsorption, salt loss syndromes, male infertility and leading…”
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Therapeutic pipeline for individuals with cystic fibrosis with mutations nonresponsive to current cystic fibrosis transmembrane conductance regulator modulators by Fajac, Isabelle, Sermet-Gaudelus, Isabelle

“…Cystic fibrosis is a severe autosomal recessive disorder caused by mutations in the cystic fibrosis transmembrane conductance regulator gene (CFTR) encoding…”
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Emerging medicines to improve the basic defect in cystic fibrosis by Fajac, Isabelle, Sermet-Gaudelus, Isabelle

“…Cystic fibrosis (CF) is a severe autosomal recessive disorder featuring exocrine pancreatic insufficiency and bronchiectasis. It is caused by mutations in the…”
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Therapeutic pipeline for individuals with cystic fibrosis with mutations nonresponsive to current cystic fibrosis transmembrane conductance regulator modulators by Fajac, Isabelle, Sermet-Gaudelus, Isabelle

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Uptake of plasmid/glycosylated polymer complexes and gene transfer efficiency in differentiated airway epithelial cells by Fajac, Isabelle, Thévenot, Guiti, Bédouet, Laurent, Danel, Claire, Riquet, Marc, Merten, Marc, Figarella, Catherine, Ava-Santucci, Josette Dall', Monsigny, Michel, Briand, Pascale

“…Background We have studied gene transfer efficiency of glycosylated polylysines and glycosylated polyethylenimines as vectors in immortalized differentiated…”
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No easy road to better cystic fibrosis care in Eastern Europe? by De Boeck, Kris, Fajac, Isabelle

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The future of cystic fibrosis care: a global perspective by Bell, Scott C, Mall, Marcus A, Gutierrez, Hector, Macek, Milan, Madge, Susan, Davies, Jane C, Burgel, Pierre-Régis, Tullis, Elizabeth, Castaños, Claudio, Castellani, Carlo, Byrnes, Catherine A, Cathcart, Fiona, Chotirmall, Sanjay H, Cosgriff, Rebecca, Eichler, Irmgard, Fajac, Isabelle, Goss, Christopher H, Drevinek, Pavel, Farrell, Philip M, Gravelle, Anna M, Havermans, Trudy, Mayer-Hamblett, Nicole, Kashirskaya, Nataliya, Kerem, Eitan, Mathew, Joseph L, McKone, Edward F, Naehrlich, Lutz, Nasr, Samya Z, Oates, Gabriela R, O'Neill, Ciaran, Pypops, Ulrike, Raraigh, Karen S, Rowe, Steven M, Southern, Kevin W, Sivam, Sheila, Stephenson, Anne L, Zampoli, Marco, Ratjen, Felix

“…The past six decades have seen remarkable improvements in health outcomes for people with cystic fibrosis, which was once a fatal disease of infants and young…”
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Disease-specific clinical trials networks: the example of cystic fibrosis by De Boeck, Kris, Bulteel, Veerle, Fajac, Isabelle

“…This article describes the steps of the development and the structure of a disease-specific clinical trials network for cystic fibrosis in Europe. Activities…”
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Triple Therapy for Cystic Fibrosis Phe508del–Gating and –Residual Function Genotypes by Barry, Peter J, Mall, Marcus A, Álvarez, Antonio, Colombo, Carla, de Winter-de Groot, Karin M, Fajac, Isabelle, McBennett, Kimberly A, McKone, Edward F, Ramsey, Bonnie W, Sutharsan, Sivagurunathan, Taylor-Cousar, Jennifer L, Tullis, Elizabeth, Ahluwalia, Neil, Jun, Lucy S, Moskowitz, Samuel M, Prieto-Centurion, Valentin, Tian, Simon, Waltz, David, Xuan, Fengjuan, Zhang, Yaohua, Rowe, Steven M, Polineni, Deepika

“…Elexacaftor–ivacaftor–tezacaftor therapy is efficacious in cystic fibrosis among patients with at least one copy of the Phe508del allele. In this trial…”
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Cystic fibrosis by Fajac, Isabelle, Burgel, Pierre-Régis

“…Cystic fibrosis (CF) is an autosomal recessive genetic disease caused by variants in the gene encoding for the cystic fibrosis transmembrane conductance…”
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The French Compassionate Program of elexacaftor-tezacaftor-ivacaftor in people with cystic fibrosis with advanced lung disease and no F508del CFTR variant by Burgel, Pierre-Régis, Sermet-Gaudelus, Isabelle, Durieu, Isabelle, Kanaan, Reem, Macey, Julie, Grenet, Dominique, Porzio, Michele, Coolen-Allou, Nathalie, Chiron, Raphael, Marguet, Christophe, Douvry, Benoit, Dufeu, Nadine, Danner-Boucher, Isabelle, Foucaud, Pierre, Lemonnier, Lydie, Girodon, Emmanuelle, Da Silva, Jennifer, Martin, Clémence

“…The European Medicines Agency has approved the cystic fibrosis transmembrane conductance regulator (CFTR) modulator combination…”
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Non-respiratory health-related quality of life in people with cystic fibrosis receiving elexacaftor/tezacaftor/ivacaftor by Fajac, Isabelle, Daines, Cori, Durieu, Isabelle, Goralski, Jennifer L., Heijerman, Harry, Knoop, Christiane, Majoor, Christof, Bruinsma, Bote G., Moskowitz, Samuel, Prieto-Centurion, Valentin, Van Brunt, Kate, Zhang, Yaohua, Quittner, Alexandra

“…•In two previous phase 3 studies of elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA).• ELX/TEZ/IVA improved pulmonary function and CFQ-R respiratory domain…”
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