Discovery Network
Understanding cellular waste recycling
Validate therapeutic targets
£689,055.85
View projectTargeting PARP to rescue TDP-43
Dr Leeanne McGurk, University of Dundee
Advancing Treatments Award
Validate therapeutic targets
October 2026 – September 2029
£335,349.17
Summary
In most cases of MND, a protein called TDP-43 is in the wrong location in motor neurons, meaning that it can no longer perform its important functions. Dr McGurk’s team discovered that another group of proteins, called PARPs, help to keep TDP-43 in its correct location. However, PARPs themselves are overactive or misplaced in MND, which means they might be a good target for therapy development. In this project, the team aim to further understand how PARPs help TDP-43 perform its normal functions. They will assess how drugs that reduce the activity of PARPs (which are currently used to treat cancer) affect different types of brain cells. and how effective they are at entering the brain, which is often a challenge when developing drugs for neurological conditions.
Why this research is important
This research will aid our understanding of how PARPs support TDP-43 function and assess whether drugs to reduce PARP activity are likely to be safe and effective in the context of MND. These drugs are already used to treat cancer meaning a lot is already known about how they behave in the human body; as such, their use as a repurposed treatment for MND could be fast-tracked.
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