FFC#11/2021

Alternative targets for the treatment of cystic fibrosis basic defect

AREA 1 Therapies to correct the underlying defect

FFC#11/2021

Alternative targets for the treatment of cystic fibrosis basic defect
€ 0 still needed
0%
€ 104.000 goal

pRINCIPAL INVESTIGATOR

Paolo Scudieri (Dipartimento di neuroscienze, riabilitazione, oftalmologia, genetica e scienze materno-infantili – DINOGMI, Università degli Studi di Genova)

Partner

Fabiana Ciciriello (IRCSS Ospedale Pediatrico Bambino Gesù, Roma)

Researchers

5

Category

AREA 1 Therapies to correct the underlying defect

Duration

2 years

Goal

€ 104.000

Funds raised

€ 104.000

Objectives

This project is focused on the study and modulation of alternative targets to CFTR. Through various technological approaches (immunofluorescence assays and confocal microscopy), the researchers aim to evaluate the expression and function of alternative targets in the airways, in order to develop targeted therapeutic approaches. The project involves the use of airway epithelial cells obtained from persons with cystic fibrosis (CF) of various ages, genotypes and clinical conditions and healthy controls using minimally invasive procedures (brushing of the mucous membrane of the nasal cavities). The effect of potentiation or inhibition of these targets on the CF phenotype will also be tested by in vitro studies. Finally, the researchers propose to develop tools and assays for the search for new pharmacological modulators.


XIX Convention FFC Ricerca – download here a brief presentation of the project

WHO ADOPTED THE PROJECT

Gruppo di sostegno FFC Ricerca di Sondrio Tresivio Ponte “In ricordo di Teresa”

€ 20.000

Delegazione FFC Ricerca di Codogno e Piacenza

€ 8.000

Gruppo di sostegno FFC Ricerca di Magenta

€ 10.000

Charity Dinner “Respiri”

€ 40.000

Gruppo di sostegno FFC Ricerca Miriam Colombo – Ospedaletti

€ 11.000

Delegazione FFC Ricerca di Lucca

€ 15.000

OTHER PROJECTS

Discover the other projects

GMRF#1/2026

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