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TITLE: Fundamental and translational research in Cystic Fibrosis-why we still need it ?  Full Text
AUTHORS: Farinha, Carlos M.; Brodsky, Jeffrey L.; Pedemonte, Nicoletta;
PUBLISHED: 2023, SOURCE: JOURNAL OF CYSTIC FIBROSIS, VOLUME: 22
INDEXED IN: Scopus WOS
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TITLE: Mutation-class dependent signatures outweigh disease-associated processes in cystic fibrosis cells  Full Text
AUTHORS: Santos, Lucia; Nascimento, Rui; Duarte, Aires; Railean, Violeta; Amaral, Margarida D. D.; Harrison, Patrick T. T.; Gama Carvalho, Margarida; Farinha, Carlos M. M.;
PUBLISHED: 2023, SOURCE: CELL AND BIOSCIENCE, VOLUME: 13, ISSUE: 1
INDEXED IN: Scopus WOS
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TITLE: Rescue of Rare CFTR Trafficking Mutants Highlights a Structural Location-Dependent Pattern for Correction  Full Text
AUTHORS: Zacarias, Sonia; Batista, Marta S. P.; Ramalho, Sofia S.; Victor, Bruno L.; Farinha, Carlos M.;
PUBLISHED: 2023, SOURCE: INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES, VOLUME: 24, ISSUE: 4
INDEXED IN: Scopus WOS
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TITLE: Fundamental and translational research in Cystic Fibrosis - why we still need it  Full Text
AUTHORS: Farinha, Carlos M.; Brodsky, Jeffrey L.; Pedemonte, Nicoletta;
PUBLISHED: 2023, SOURCE: JOURNAL OF CYSTIC FIBROSIS, VOLUME: 22
INDEXED IN: WOS
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TITLE: Use of adenine base editing and homology-independent targeted integration strategies to correct the cystic fibrosis causing variant, W1282X
AUTHORS: Mention, Karen; Cavusoglu Doran, Kader; Joynt, Anya T.; Santos, Lucia; Sanz, David; Eastman, Alice C.; Merlo, Christian; Langfelder Schwind, Elinor; Scallan, Martina F.; Farinha, Carlos M.; Cutting, Garry R.; Sharma, Neeraj; Harrison, Patrick T.;
PUBLISHED: 2023, SOURCE: HUMAN MOLECULAR GENETICS, VOLUME: 32, ISSUE: 23
INDEXED IN: Scopus WOS
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TITLE: Rescue of Mutant CFTR Trafficking Defect by the Investigational Compound MCG1516A  Full Text
AUTHORS: Lopes Pacheco, M; Bacalhau, M; Ramalho, SS; Silva, IAL; Ferreira, FC; Carlile, GW; Thomas, DY; Farinha, CM; Hanrahan, JW; Amaral, MD;
PUBLISHED: 2022, SOURCE: CELLS, VOLUME: 11, ISSUE: 1
INDEXED IN: Scopus WOS
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TITLE: Rare Trafficking CFTR Mutations Involve Distinct Cellular Retention Machineries and Require Different Rescuing Strategies  Full Text
AUTHORS: Ramalho, SS; Silva, IAL; Amaral, MD; Farinha, CM;
PUBLISHED: 2022, SOURCE: INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES, VOLUME: 23, ISSUE: 1
INDEXED IN: WOS
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TITLE: Comparison of Cas9 and Cas12a CRISPR editing methods to correct the W1282X-CFTR mutation  Full Text
AUTHORS: Santos, L; Mention, K; Cavusoglu Doran, K; Sanz, DJ; Bacalhau, M; Lopes Pacheco, M; Harrison, PT; Farinha, CM;
PUBLISHED: 2022, SOURCE: JOURNAL OF CYSTIC FIBROSIS, VOLUME: 21, ISSUE: 1
INDEXED IN: WOS
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TITLE: Severity of the S1251N allele in cystic fibrosis is affected by the presence of the F508C variant in cis  Full Text
AUTHORS: Cuyx, Senne; Ramalho, Sofia S.; Callebaut, Isabelle; Cuppens, Harry; Kmit, Arthur; Arnauts, Kaline; Ferrante, Marc; Verfaillie, Catherine; Ensinck, Marjolein; Carlon, Marianne S.; Boon, Mieke; Proesmans, Marijke; Dupont, Lieven; De Boeck, Kris; Farinha, Carlos M.; Vermeulen, Francois; Ramalho, Anabela S.;
PUBLISHED: 2022, SOURCE: JOURNAL OF CYSTIC FIBROSIS, VOLUME: 21, ISSUE: 4
INDEXED IN: Scopus WOS
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