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CFTR modulators response of S737F and T465N CFTR variants on patient-derived rectal organoids

Abstract Background Predictions based on patient-derived materials of CFTR modulators efficacy have been performed lately in patient-derived cells, extending FDA-approved drugs for CF patients harboring rare variants. Here we developed intestinal organoids from subjects carrying S737F- and T465N-CFT...

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Váldodahkkit: Karina Kleinfelder, Paola Melotti, Anca Manuela Hristodor, Cristina Fevola, Giovanni Taccetti, Vito Terlizzi, Claudio Sorio
Materiálatiipa: Artigo
Giella:Inglês
Almmustuhtton: BMC 2024-09-01
Ráidu:Orphanet Journal of Rare Diseases
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Liŋkkat:https://doi.org/10.1186/s13023-024-03334-3
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