FFC#7/2020

Functional role of post-translational modifications in F508del-CFTR correction

AREA 1 Therapies to correct the underlying defect

FFC#7/2020

Functional role of post-translational modifications in F508del-CFTR correction
€ 0 still needed
0%
€ 40.000 goal

pRINCIPAL INVESTIGATOR

Mauro Salvi (Università degli Studi di Padova, Dip. di Scienze Biomediche)

Researchers

3

Category

AREA 1 Therapies to correct the underlying defect

Duration

1 year

Goal

€ 40.000

Funds raised

€ 40.000

Objectives

This is the extension of the FFC#11/2019 pilot project; it aims at discovering what are the modifications of the CFTR protein during the maturation process which takes place after its synthesis is carried out. In fact, the F508del mutant protein’s functional recovery induced by correctors is associated with a number of modifications, called “post-translational” modifications, which are a characteristic of the normal protein. The main objective is to clarify the real contribution of the post-translational modifications in the functional recovery of F508del. For this purpose, 5 enzymes, called “demethylases”, have been identified, which are involved in the F508del’s maturation induced by correctors by acting on protein’s methylation (addition or subtraction of the methyl group CH3). The study intends to understand whether it is possible to promote the recovery of the mutated channel by controlling those changes, with particular reference to protein methylation.

WHO ADOPTED THE PROJECT

Delegazione FFC di Boschi Sant’Anna Minerbe “Alla fine esce sempre il sole”

€ 40.000

Delegazione FFC di Catania Mascalucia

€ 21.500

Delegazione FFC di Vigevano

€ 47.000

OTHER PROJECTS

Discover the other projects

GMSG#1/2025

Developing PNAs to Block Essential Bacterial Genes and Create New Antimicrobial Therapies

FFC#1/2025

Understanding the process of epithelial-mesenchymal transition to preserve tissue function and reduce long-term complications (e.g. cancer)

FFC#2/2025

Developing RNA-targeted therapies to regulate gene expression and restore CFTR synthesis