INTESTINAL Publication-derived

CRISPR Correction of CFTR F508del in Patient Organoids

Source Schwank et al. · Hubrecht Institute, Utrecht, Netherlands

👤 Schwank G, Koo BK, Sasselli V, Dekkers JF, Heo I, Demircan T, Sasaki N, Boymans S, Cuppen E, van der Ent CK, Nieuwenhuis EES, Beekman JM, Clevers H ⏱ 20160 min 🧫 Primary (Human CF Patient Intestinal Organoids, F508del/F508del)

Abstract

Achieved the first CRISPR-Cas9 correction of the CFTR F508del mutation in patient-derived intestinal organoids, restoring functional CFTR chloride channel activity as demonstrated by forskolin-induced swelling. This landmark proof-of-concept established organoid gene therapy feasibility.

Cell source

Primary (Human CF Patient Intestinal Organoids, F508del/F508del)

Application

Gene Therapy Development

Full SOP

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Attribution

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

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