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

Development of drug based therapies to target rare mutations causing severe cystic fibrosis

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

In this project, we will focus on identifying novel CFTR modulators that can rescue CFTR trafficking to
the plasma membrane of the N1303K and G85E mutations. N1303K is the second most frequent
mutation in Belgium with a worldwide prevalence of around 3%. G85E occurs in Belgian patients with
Mediterranean ancestry and has a worldwide prevalence of 0.8%. This project builds on the combined
expertise of Dr. Carlon’s team on viral vector technology for cystic fibrosis in cell and animal models,
including patient derived intestinal organoids, and Prof Debyser’s group on drug discovery as shown by
successes in other disease areas. The project is innovative as it will provide further insights in the
functional defect of the N1303K and G85E mutations and lead to novel personalized treatment
strategies for a subset of CF patients, which are not in direct focus of pharma industry, yet suffer from a
severe disease phenotype. Impact will reach beyond the N1303K and G85E mutations as strategies
applied and assays developed guide us and others to implement similar strategies for other patient
populations.

Institute: KU Leuven
City: Leuven
Project amount (€): 200.000

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