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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| Hauptverfasser: | , , , , , , |
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| Format: | Artigo |
| Sprache: | Inglês |
| Veröffentlicht: |
BMC
2024-09-01
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| Schriftenreihe: | Orphanet Journal of Rare Diseases |
| Schlagworte: | |
| Online-Zugang: | https://doi.org/10.1186/s13023-024-03334-3 |
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