FFC#2/2016

Alternative strategies for F508del-CFTR repair: novel targets for drug discovery approach in cystic fibrosis

FFC#2/2016

Alternative strategies for F508del-CFTR repair: novel targets for drug discovery approach in cystic fibrosis

PRINCIPAL INVESTIGATOR

Giorgio Cozza (Istituto Europeo per Ricerca in Fibrosi Cistica-IERFC presso Divisione di Genetica e Biologia Cellulare, Istituto San Raffaele, Milano)

Partner

Antonella Tosco (Dipartimento di Scienze Mediche Traslazionali, Università di Napoli Federico II, Centro Regionale Fibrosi Cistica)

RESEARCHERS

11

CATEGORY

AREA 1 Therapies to correct the underlying defect

DURATION

1 year

GOAL

€ 40.000 €

RESULTS

The chemical optimization of cysteamine led to a novel compound (CT11), active in restoring CFTR-F508delfunction equally to cysteamine in cell models (CFBE41o-), but at a concentration 500 fold lower. These results were confirmed also in ex vivo experiments in nasal cells collected by nasal brushing from CF patients and in CFTR-F508del homozygous mice. Further, researchers carried out a selection of natural compounds and obtained the discovery of a three natural molecules (AC1, AC2, AC3) able to restore between 50-78% of CFTR-F508del function in cell models (CFBE41o-) and novel PDI inhibitors active in restoring CFTR-F508del function. Three compounds (SPH1, SPH2 and SEC1) with PDI inhibitory activity shew promising characteristics in restoring CFTR-F508del function in cell models. The project FFC#10/2017 will exploit essentially the same methodology previously described with the aim to develop a novel generation of optimized compounds against TG2, PDI and protein kinases.

OTHER RESULTS

FFC #3/2024

Two molecules are effective in activating Heat Shock Proteins and enhancing the action of CFTR correctors with the F508del mutation in vitro.

FFC#5/2024

Some peptide nucleic acids (PNAs) re-sensitise Pseudomonas aeruginosa to the antibiotic meropenem in vitro and reduce its virulence.

FFC#1/2023

Tezacaftor, one of the components of Kaftrio, induces an accumulation of dihydroceramides both in vitro and in vivo in animal models