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Discovery of CFTR modulators for the treatment of cystic fibrosis

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Pages 897-913 | Received 30 Nov 2020, Accepted 31 Mar 2021, Published online: 13 Apr 2021

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Kathryn E. Oliver, Marianne S. Carlon, Nicoletta Pedemonte & Miquéias Lopes-Pacheco. (2023) The revolution of personalized pharmacotherapies for cystic fibrosis: what does the future hold?. Expert Opinion on Pharmacotherapy 24:14, pages 1545-1565.
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Madalena C Pinto, Iris A L Silva, Miriam F Figueira, Margarida D Amaral & Miquéias Lopes-Pacheco. (2021) Pharmacological Modulation of Ion Channels for the Treatment of Cystic Fibrosis. Journal of Experimental Pharmacology 13, pages 693-723.
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Mafalda Bacalhau, Mariana Camargo, Grace A. V. Magalhães-Ghiotto, Sybelle Drumond, Carlos Henrique M. Castelletti & Miquéias Lopes-Pacheco. (2023) Elexacaftor-Tezacaftor-Ivacaftor: A Life-Changing Triple Combination of CFTR Modulator Drugs for Cystic Fibrosis. Pharmaceuticals 16:3, pages 410.
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Anabela S. Ramalho, Felice Amato & Martina Gentzsch. (2023) Patient-derived cell models for personalized medicine approaches in cystic fibrosis. Journal of Cystic Fibrosis 22, pages S32-S38.
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Sacha Spelier, Eyleen de Poel, Georgia N. Ithakisiou, Sylvia W.F. Suen, Marne C. Hagemeijer, Danya Muilwijk, Annelotte M. Vonk, Jesse E. Brunsveld, Evelien Kruisselbrink, Cornelis K. van der Ent & Jeffrey M. Beekman. (2023) High-throughput functional assay in cystic fibrosis patient-derived organoids allows drug repurposing. ERJ Open Research 9:1, pages 00495-2022.
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Anya T. Joynt, Garry R. Cutting & Neeraj Sharma. (2022) Genetics of Cystic Fibrosis. Clinics in Chest Medicine 43:4, pages 591-602.
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Katelin M. Allan, Miro A. Astore, Laura K. Fawcett, Sharon L. Wong, Po-Chia Chen, Renate Griffith, Adam Jaffe, Serdar Kuyucak & Shafagh A. Waters. (2022) S945L-CFTR molecular dynamics, functional characterization and tezacaftor/ivacaftor efficacy in vivo and in vitro in matched pediatric patient-derived cell models. Frontiers in Pediatrics 10.
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Alyssa Bowling, Alice Eastman, Christian Merlo, Gabrielle Lin, Natalie West, Shivani Patel, Garry Cutting & Neeraj Sharma. (2022) Downstream Alternate Start Site Allows N-Terminal Nonsense Variants to Escape NMD and Results in Functional Recovery by Readthrough and Modulator Combination. Journal of Personalized Medicine 12:9, pages 1448.
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Chiara Papi, Jessica Gasparello, Matteo Zurlo, Alex Manicardi, Roberto Corradini, Giulio Cabrini, Roberto Gambari & Alessia Finotti. (2022) Combined Treatment of Bronchial Epithelial Calu-3 Cells with Peptide Nucleic Acids Targeting miR-145-5p and miR-101-3p: Synergistic Enhancement of the Expression of the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Gene. International Journal of Molecular Sciences 23:16, pages 9348.
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Iris A. L. Silva, Onofrio Laselva & Miquéias Lopes-Pacheco. (2022) Advances in Preclinical In Vitro Models for the Translation of Precision Medicine for Cystic Fibrosis. Journal of Personalized Medicine 12:8, pages 1321.
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John A. King, Anna-Louise Nichols, Sian Bentley, Siobhan B. Carr & Jane C. Davies. (2022) An Update on CFTR Modulators as New Therapies for Cystic Fibrosis. Pediatric Drugs 24:4, pages 321-333.
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Tamorah Lewis, Kelly C. Wade & Jonathan M. Davis. (2022) Challenges and opportunities for improving access to approved neonatal drugs and devices. Journal of Perinatology 42:6, pages 825-828.
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Amanda Centorame, Daciana Catalina Dumut, Mina Youssef, Martin Ondra, Irenej Kianicka, Juhi Shah, Radu Alexandru Paun, Tomas Ozdian, John W. Hanrahan, Ekaterina Gusev, Basil Petrof, Marian Hajduch, Radu Pislariu, Juan Bautista De Sanctis & Danuta Radzioch. (2022) Treatment With LAU-7b Complements CFTR Modulator Therapy by Improving Lung Physiology and Normalizing Lipid Imbalance Associated With CF Lung Disease. Frontiers in Pharmacology 13.
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Fabiana Ciciriello, Marcel J. C. Bijvelds, Federico Alghisi, Kelly F. Meijsen, Luca Cristiani, Claudio Sorio, Paola Melotti, Alessandro G. Fiocchi, Vincenzina Lucidi & Hugo R. De Jonge. (2022) Theratyping of the Rare CFTR Variants E193K and R334W in Rectal Organoid-Derived Epithelial Monolayers. Journal of Personalized Medicine 12:4, pages 632.
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Miquéias Lopes-Pacheco, Mafalda Bacalhau, Sofia S. Ramalho, Iris A. L. Silva, Filipa C. Ferreira, Graeme W. Carlile, David Y. Thomas, Carlos M. Farinha, John W. Hanrahan & Margarida D. Amaral. (2022) Rescue of Mutant CFTR Trafficking Defect by the Investigational Compound MCG1516A. Cells 11:1, pages 136.
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Ana M. Matos, Peter Jordan & Paulo Matos. (2021) Treatment of Polarized Cystic Fibrosis Airway Cells With HGF Prevents VX-661-Rescued F508del-CFTR Destabilization Caused by Prolonged Co-exposure to VX-770. Frontiers in Molecular Biosciences 8.
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Carla M. P. Ribeiro & Martina Gentzsch. (2021) Impact of Airway Inflammation on the Efficacy of CFTR Modulators. Cells 10:11, pages 3260.
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Guido Veit, Tony Velkov, Haijin Xu, Nathalie Vadeboncoeur, Lara Bilodeau, Elias Matouk & Gergely Lukacs. (2021) A Precision Medicine Approach to Optimize Modulator Therapy for Rare CFTR Folding Mutants. Journal of Personalized Medicine 11:7, pages 643.
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