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 |