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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Manylion Llyfryddiaeth

Prif Awduron:	Karina Kleinfelder, Paola Melotti, Anca Manuela Hristodor, Cristina Fevola, Giovanni Taccetti, Vito Terlizzi, Claudio Sorio
Fformat:	Artigo
Iaith:	Inglês
Cyhoeddwyd:	BMC 2024-09-01
Cyfres:	Orphanet Journal of Rare Diseases
Pynciau:	Human intestinal monolayers CFTR modulators Electrophysiological measurements Rare mutations Personalized medicine
Mynediad Ar-lein:	https://doi.org/10.1186/s13023-024-03334-3
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