FFC#7/2016

Human intestinal organoids for detecting CFTR rescue molecules in human plasma samples

AREA 1 Therapies to correct the underlying defect

FFC#7/2016

Human intestinal organoids for detecting CFTR rescue molecules in human plasma samples
€ 0 still needed
0%
€ 35.000 goal

pRINCIPAL INVESTIGATOR

Paola Melotti (Centro Fibrosi Cistica, Azienda Ospedaliera Universitaria Integrata di Verona)

Researchers

6

Category

AREA 1 Therapies to correct the underlying defect

Duration

2 years

Goal

€ 35.000

Funds raised

€ 35.000

Objectives

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

OTHER PROJECTS

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Exploring the role of PKD1 in promoting CFTR stability and function at the cell surface

FFC#1/2026

Mapping proteins regulating CFTR mRNA stability to identify new therapeutic targets for nonsense mutations

FFC#2/2026

Evaluating the potential of phosphodiesterase inhibitors to enhance the efficacy of CFTR modulators and support personalized therapeutic approaches