FFC#1/2014

Identification and validation of novel molecules obtained by integrated computational and experimental approaches for the read-through of PTCs in CF cells

AREA 1 Therapies to correct the underlying defect

FFC#1/2014

Identification and validation of novel molecules obtained by integrated computational and experimental approaches for the read-through of PTCs in CF cells
€ 0 still needed
0%
€ 38.000 goal

pRINCIPAL INVESTIGATOR

Laura Lentini (Dip. di Biologia, Scienze Chimiche e Farmaceutiche e Tecnologie -STEBICEF, Sez. di Biologia Cellulare, Università di Palermo)

Partner

Ivana Pibiri (Dip. di Biologia, Scienze Chimiche e Farmaceutiche e Tecnologie – STEBICEF, Sez. di Biologia Cellulare, Università di Palermo)

Researchers

7

Category

AREA 1 Therapies to correct the underlying defect

Duration

2 years

Goal

€ 38.000

Funds raised

€ 38.000

Objectives

The efficacy of novel small molecules derivatives of the PTC124 previously identified will be evaluated in CF cells (primary human bronchial epithelial cells with G542X mutation and in immortalized epithelial cells of cystic fibrosis patients). Moreover new molecules will be selected by a virtual screening based on “in house” and available public compound libraries; then lead compounds will be designed and synthesized. The mechanism of action of PTC’s read-through promoters will be investigated: molecular dynamics and docking studies aimed to model the interaction between the bioactive synthesized compounds and their possible cellular target will be carried out.

WHO ADOPTED THE PROJECT

Delegazione FFC di Palermo, Delegazione FFC di Vittoria Ragusa e Siracusa, Delegazione FFC di Catania Mascalucia

€ 38.000

LIFC Ass. Siciliana in ricordo di Davide Radicello

€ 20.000

Danone Italia

€ 50.000

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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