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INTESTINAL Publication-derived

Organoid-Based Prediction of Ivacaftor Response for Rare CFTR

Source Dekkers et al. · University Medical Centre Utrecht and Erasmus MC, Netherlands

👤 Dekkers JF, Berkers G, Kruisselbrink E, Vonk A, de Jonge HR, Janssens HM, Bronsveld I, van de Graaf EA, Nieuwenhuis EES, Houwen RHJ, Vleggaar FP, Escher JC, de Rijke YB, Majoor CJ, Heijerman HGM, de Winter-de Groot KM, Clevers H, van der Ent CK, Beekman JM ⏱ 6720 min 🧫 Primary (Human Rectal Biopsies from CF Patients with Rare CFTR Genotypes)

Abstract

Demonstrated that organoid forskolin-induced swelling predicts clinical ivacaftor response for rare CFTR mutations lacking clinical trial data. This paradigm enables drug access through compassionate use programs and has been accepted for reimbursement decisions in the Netherlands.

Cell source
Primary (Human Rectal Biopsies from CF Patients with Rare CFTR Genotypes)
Application
Rare Mutation Drug Access

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Attribution

This SOP was authored by Organthis based on the published method in Dekkers et al.. The originating laboratory holds no rights in this SOP and has not endorsed it unless marked Verified.

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