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Science (New York, N.Y.)|June 13, 2024
Gene editing flows to the lungsMattijs Bulcaen, Marianne S CarlonProgress in Molecular Biology and Translational Science|June 27, 2021
Rewriting CFTR to cure cystic fibrosisGiulia Maule, Marjolein Ensinck, Mattijs Bulcaen, et al.International Journal of Molecular Sciences|May 7, 2025
Cis-Regulation of the CFTR Gene in Pancreatic CellsClara Blotas, Anaïs Le Nabec, Mégane Collobert, et al.STAR Protocols|February 2, 2025
Protocol for functional screening of CFTR-targeted genetic therapies in patient-derived organoids using DETECTOR deep-learning-based analysisMattijs Bulcaen, Ronald B Liu, Kasper Gryspeert, et al.Science Translational Medicine|April 22, 2026
Functional correction of the untreatable CFTR 1717-1G>A mutation through mRNA- and sgRNA-optimized base editingAlessandro Umbach, Annalisa Santini, Mattijs Bulcaen, et al.Cells|June 24, 2022
One Size Does Not Fit All: The Past, Present and Future of Cystic Fibrosis Causal TherapiesMarjolein M Ensinck, Marianne S CarlonPrenatal Diagnosis|October 6, 2017
Roadmap for an early gene therapy for cystic fibrosis airway diseaseMarianne S Carlon, Dragana Vidović, Susan BirketHuman Gene Therapy|April 28, 2025
Trojan Horse-Like Vehicles for CRISPR-Cas Delivery: Engineering Extracellular Vesicles and Virus-Like Particles for Precision Gene Editing in Cystic FibrosisLaudonia Lidia Dipalo, Jacob Giehm Mikkelsen, Rik Gijsbers, et al.Expert Opinion on Pharmacotherapy|June 28, 2023
The revolution of personalized pharmacotherapies for cystic fibrosis: what does the future hold?Kathryn E Oliver, Marianne S Carlon, Nicoletta Pedemonte, et al.Cell Reports. Medicine|May 2, 2024
Prime editing functionally corrects cystic fibrosis-causing CFTR mutations in human organoids and airway epithelial cellsMattijs Bulcaen, Phéline Kortleven, Ronald B Liu, et al.Pageof 4