FFC#3/2018

Dissecting the rescue mechanisms mediated by CFTR correctors

AREA 1 Therapies to correct the underlying defect

FFC#3/2018

Dissecting the rescue mechanisms mediated by CFTR correctors
€ 0 still needed
0%
€ 50.000 goal

pRINCIPAL INVESTIGATOR

Debora Baroni (Istituto di Biofisica, CNR, Genova)

Researchers

5

Category

AREA 1 Therapies to correct the underlying defect

Duration

2 years

Goal

€ 50.000

Funds raised

€ 50.000

Objectives

The project of these biophysicists of the CNR of Genoa intends to continue the important studies conducted with FFC funding and aimed at better understanding the mechanisms of maturation, folding and trafficking of the CFTR protein, normal and altered due to the F508del mutation. Some correctors of mutated CFTR have been the subject of clinical studies that have highlighted some therapeutic benefits but also many limitations. Therefore, the need to identify new and more effective correctors becomes ever more pressing. A better understanding of the mechanism of action of the existing ones, as well as the understanding of the basic defect caused by the F508del mutation, they will allow a significant step forward in the treatment of the disease. The main objective of the project is to identify the domains (parts of the protein) and the sub-domains of mutated CFTR involved in the recovery action of defects of folding and ripening mediated by the correctors, with the aim of reaching information that allows the design of more effective correctors.

WHO ADOPTED THE PROJECT

Delegazione FFC Valle Scrivia Alessandria

€ 8.000

Gruppo di Sostegno FFC di Genova “Mamme per la Ricerca”

€ 42.000

Matilde e Paola per Elsa

€ 10.000

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