FFC#11/2018

Rescuing defective CFTR applying a drug repositioning strategy based on computational studies, surface plasmon resonance and cell-based assays

AREA 1 Therapies to correct the underlying defect

FFC#11/2018

Rescuing defective CFTR applying a drug repositioning strategy based on computational studies, surface plasmon resonance and cell-based assays
€ 0 still needed
0%
€ 39.000 goal

pRINCIPAL INVESTIGATOR

Marco Rusnati (Università degli Studi di Brescia, Dip. Medicina Molecolare e Traslazionale, Sez. Oncologia e Immunologia)

Partner

Paola Fossa (Università degli Studi di Genova, Dip. di Farmacia); Alessandro Orro (Istituto di Tecnologie biomediche, CNR, Milano)

Researchers

11

Category

AREA 1 Therapies to correct the underlying defect

Duration

1 year

Goal

€ 39.000

Funds raised

€ 39.000

Objectives

Another project that searches for alternative ways to overcome the limits of CFTR modulators currently in use or under study. It aims to evaluate a wide range of drugs and natural products already in use for other therapies with the intent to identify those that could have significant effect in the recovery of the CFTR-F508del protein. The repositioning of known drugs, with the advantage of not having to travel the long and costly way of inventing new ones, has already allowed the discovery of new drugs for different diseases and so it can be for CF. The study will make use of a dual technological approach: bioinformatics and “surface plasmon resonance” (SPR). This will also allow to characterize the mechanism of action of small therapeutic molecules for CF. The project will proceed with the use of appropriate CF cell models (including primary cultures from CF bronchi) to validate the therapeutic potential of drugs selected in the first instance as active in the recovery of CFTR. Repositionable CF drugs will be considered by the clinical advisor for comparison and compatibility with existing cystic fibrosis drugs.

WHO ADOPTED THE PROJECT

Delegazione FFC di Torino

€ 39.000

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