Investigating if mechanisms involving TDP-43 could be targets for therapeutic intervention.
Principal Investigator: Prof Jernej Ule
Lead Institution: King's College London
MND Association Funding: £298,133
Funding dates: December 2022 - November 2025
About the project
TDP-43 protein clumps in neurons are a feature of nearly all cases of MND, making this protein a key target for the development of new therapies. However, as TDP-43 has many different roles within neurons, it is very difficult to target this protein. Several mechanisms that normally control the properties of the TDP-43 protein have been identified and these help to maintain the function and amount of TDP-43. Mutations in TDP-43 that cause MND might be interrupting these mechanisms and causing clumps of the protein to form. This project will use cell models of MND to look at whether these mechanisms could be promising therapeutic targets. It also aims to identify new therapeutic strategies to restore the mechanisms and limit the formation of TDP-43 clumps.
What could this mean for MND research?
This project will try to find new potential targets for therapeutic intervention. Once found, the researchers will try to see if they can prevent or correct the damage being done. This work could help to find potential new treatments in cases of MND which have TDP-43 protein clumps.
Project code: 886-791