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

Mechanism of action of new correctors of the F508del mutation

CFTR
New Therapies
Basic research Netherlands Prof. Jeffrey Beekman
Start date: 2017
End date: 2021
Status: Ongoing
Abstract:

This research aims to better understand how the new substances of Galapagos work on the defective CFTR, using mini-intestines (organoids) and cells from the nasal mucosa (nasal epithelial cells). Depending on the CFTR defect 1, 2 or a combination of 3 substances are required to show a clear effect. A better understanding of these reactions in the mini-arms and the nasal mucosa will help to better understand the variation in later use by the patient and provide the opportunity to give ‘person-directed medication’.

Website URL: https://www.ncfs.nl/onderzoek/lopend/hitcf
Researcher: Dr. Marne Hagemeier
Institute: UMC Utrecht
City: Utrecht
Project amount (€): 150.000

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