Human intestinal organoids could be useful for assessing the efficacy of new therapies, and provide personalized therapies for CF subjects; they also represent a permanent sample for checking CFTR activity. They can be defined as miniaturized organs; when the cells that constitute the organoids contain a functional CFTR protein, the organoids swell by induction with forskolin. Vice versa, when CFTR is not working properly, organoids remain flat-shaped. On the basis of those observations, the project aims at developing a personalized medicine approach in CF patients by collecting, expanding, storing and analyzing human intestinal organoids. Crypts will be isolated and cultured from human intestinal biopsies following established protocols already set in the laboratory. Researchers will measure CFTR activity by forskolin induced swelling (FIS) assay. Established functional tests (Gibson-Cooke and FEV1 tests) will be recorded before the treatment and at indicated times after treatment. Patients with indication of treatment with Ivacaftor and Orkambi will be tested. The expectation is measuring a variable swelling rate in CF organoids, that correlates with the outcome of the other established laboratory and clinical parameters, with the aim of developing a personalized pharmacological approach to emerging therapies.
WHO ADOPTED THE PROJECT
€ 36.000
€ 31.000