Advances in Research on Treating Cystic Fibrosis

A genetic mutation causing the disease

We have known since 1989 that cystic fibrosis affects over 100 000 people worldwide. This disease is caused by a genetic mutation that changes the CFTR protein responsible for regulating the transport of chloride through cell membranes. This malfunction leads to more viscous mucus that accumulates in the respiratory and digestive tracts.

Over 100.000 people worldwide are affected by Cystic Fibrosis.

Analysing the origins of the mutations of the CFTR protein

Prof. Govaerts and his research team from ULB have been concentrating their efforts on the faulty protein that causes cystic fibrosis. Indeed, current therapeutic approaches do not apply to a number of mutations in the functional domain of the CFTR protein. Their aim is to open new therapeutic avenues for patients bearing such rarer mutations.

The Work

The research work of Prof. Govaerts' team is divided into 3 axes:

• Studying the reasons for the mutation-induced misfolding of CFTR and whether an alternative conformation is a structural intermediate for misfolding. Indeed, this phenomenon is a physical process when the protein gains its functional tridimensional structure
• Using nanobodies to decipher the conformational network within the CFTR functional domain and establish the relation to physiopathology, meaning a relation between the protein’s structure and the cause of the disease
• Investigating the conformational effect of rare Cystic Fibrosis-causing mutations

The Foundation Renews Its Support

Having been involved since 2013 in the effort to identify and characterize nanobodies, the Air Liquide Foundation is supporting ULB researchers during this third phase focusing on the study of the mutations of the CFTR protein. A new grant of 100 000€ is being provided for the salaries of the researchers and the laboratory equipment for the 2021-2023 support.