To study the swelling of human intestinal organoids in response to different plasma levels of new mutation-targetted drugs; to correlate the entity of the swelling with the respiratory function test variations; to investigate the reproducibility of results in cryopreserved samples. Drugs active on specific mutations in the CFTR gene will be tested: Ataluren, ivacaftor and the combination of ivacaftor and lumacaftor. The model of organoids would thus be fully exploited for prediction of clinical response and monitoring of established therapies in individual patients.
WHO ADOPTED THE PROJECT
Delegazione FFC di Belluno con i Rocciatori di Fonzaso
€ 35.000
Delegazione FFC di Verbania e VCO
€ 12.000
Delegazione FFC di Bologna
€ 65.000