Project name: Project category: Kind of research: Country: Principle investigator:

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

New Therapies
Basic research Italy Prof. Giorgio Cozza
Status: Ongoing
Abstract:

The aim of the project is to target the derailed CF intracellular environment instead of directly the mutant CFTR protein. The TG2 inhibitor cysteamine (that restores disabled autophagy) and the kinase inhibitor EGCG epigallocatechin-gallate have shown very promising results in either rescuing and stabilizing F508del-CFTR at plasmatic membrane. The project will be carried out by: A) in silico approaches to identify novel chemical entities able to interact with our new protein targets; B) in vitro and in cell methodologies to validate the best candidates from (A); C) in vivo validation into appropriate CF cells and animal models.

City: Milan
Project amount (€): 200.000

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