Targeting TDP-43

Summary

TDP-43 is a protein that plays an important role in the assembly line that converts information stored in our DNA into molecules called RNA, which are used as the template for making the proteins within our cells. While TDP-43 is known to be dysregulated in about 97% of people living with MND, we still don’t have an effective way to target it therapeutically. 

Professor Baker is investigating two groups of compounds that have shown promise in reducing TDP-43 toxicity in cellular models of disease, and is now testing them in animal models of disease.

Why this research is important

TDP-43 pathology is a major hallmark of MND. If high quality pre-clinical data from animal models of disease suggests that these new compounds are safe and effective, it would position them for human clinical trials. If successful, these treatments could potentially be used to treat the majority of people living with MND.

To find out more about TDP-43, check out our infographic.

*This project is part of the Motor Neuron Disease Translational Research Fund and is co-funded by LifeArc and MND Association. Total funding of this grant is £840,298.63.