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

Development of CFTR-stabilizers as therapeutics for cystic fibrosis

New Therapies
CFTR
Basic research Belgium Dr. Cédric Govaerts
Start date: 01/07/2017
End date: 30/06/2020
Status: Ongoing
Abstract:

For most patients, Cystic Fibrosis is due to a mutation in the CFTR gene that leads to a
destabilization of the CFTR chloride channel, which is then unable to fulfil its biological role in
several organs, such as the lungs or the digestive system. In order to counteract the destabilizing
effect of the mutation, we had previously developed stabilizing nanobodies, which are mini
antibodies obtained by vaccinating lamas. By studying these nanobodies we have identified the
molecular mechanisms that permit this stabilization of CFTR. We now propose to use this new
knowledge to develop a novel strategy in order to develop stabilizing compounds that will acts as
drugs against CF.

Institute: Université Libre de Bruxelles
City: Brussels
Project amount (€): 197.000

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