FFC#4/2020

Characterization of CFTR modulators mechanism of action via Photo-Affinity Labeling (PAL) approach

AREA 1 Therapies to correct the underlying defect

FFC#4/2020

Characterization of CFTR modulators mechanism of action via Photo-Affinity Labeling (PAL) approach
€ 0 still needed
0%
€ 75.000 goal

pRINCIPAL INVESTIGATOR

Fabio Bertozzi (Istituto Italiano di Tecnologia, IIT – D3 – Chimica Farmaceutica)

Researchers

4

Category

AREA 1 Therapies to correct the underlying defect

Duration

2 years

Goal

€ 75.000

Funds raised

€ 75.000

Objectives

The research group involved in the present study collaborated in the discovery of the CFTR-F508del corrector named ARN23765, as part of the strategic project FFC Task Force for Cystic Fibrosis. The group now intends to study the specific targets and the mechanism of action of ARN23765, working in primary respiratory cells from CF patients, through a new and sophisticated method, namely PAL (Photo-affinity labeling): chemical probes, which are structurally similar to ARN23765, will be synthesized; they will include two chemical groups: the first one is photoreactive, it can be activated by UV radiation and it is capable of interacting with the molecules which are close to the location of the corrector; the second one can bind a molecular marker, that will detect the specific molecules ARN23765 interacts with. Project results will help to elucidate the molecular basis of the corrector-induced CFTR’s functional recovery. This could pave the way for the identification of new molecular targets for the treatment of CF and the design of compounds with better activity and safety features.

WHO ADOPTED THE PROJECT

Delegazione FFC di Vicenza

€ 75.000

Fondazione Corrado e Bruno Maria Zaini

€ 42.000

Delegazione FFC di Vigevano

€ 47.000

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