Testing and developing intrabodies which could be used to make faulty proteins linked to MND work better.
Principal Investigator: Dr Gareth Wright
Lead Institution: University of Essex
MND Association Funding: £332,188
Funding dates: October 2025 - September 2028
About the project
Antibodies are proteins that play an important role in the immune system. They interact with ‘foreign proteins’, for example bacteria, to help them to be removed from the body. Synthetic antibodies can be generated in the lab and used to detect specific proteins in samples, but their large size means they cannot easily enter cells, which limits their usefulness in cell models of MND and in the body. In this project, the researchers have developed a system where they can create intrabodies, which are smaller simplified molecules based on antibodies. The researchers will develop new intrabodies that interact with small changes in key proteins linked to MND, including TDP43, FUS, SOD1 and C9orf72. They will test them to see if they can make the faulty proteins work better – e.g. by keeping them in the right place within the cell, preventing them from forming harmful clumps and preventing them from affecting the overall health of the cells. Those that show the most promise will then be tested in mouse models.
What could this mean for MND research?
The aim of this work is to create new biological molecules that may be delivered by gene therapy methods as a treatment for MND. By investigating many intrabodies at the same time and comparing them, it gives the researchers the best chance of finding the most effective ones that might be beneficial for people with MND.
Project code: 2472-791
