Advancing Treatments Award
Targeting PARP to rescue TDP-43
Validate therapeutic targets
£335,349.17
View projectUnderstanding cellular waste recycling
Dr Owen Peters, Dr Uroosa Chughtai, Professor Valentina Escott-Price, Dr Natalie Connor-Robson, Dr Claudia Manzoni, Professor Patrick Lewis & Professor Alfredo Iacoangeli, Cardiff University, University College London, Royal Veterinary College & King’s College London
Discovery Network
Validate therapeutic targets
October 2026 – September 2029
£689,055.85
Summary
All cells in our body rely on an internal waste recycling system, the endo-lysosomal pathway, to stay healthy and perform their functions efficiently. In MND, this waste recycling system has been shown to stop working properly in motor neurons, thus possibly playing a key role in disease onset and progression.
The team will first identify which genetic mutations found in people with MND are linked to the endo-lysosomal pathway. They will then assess the therapeutic potential of these specific mutations by introducing them in patient-derived motor neurons in a dish and investigating their effects on waste recycling and overall cellular health. This will generate in-depth understanding of the importance of the endo-lysosomal pathway in MND and explore its potential role to unlock new treatments.
Why this research is important
The endo-lysosomal pathway is a key biological component of our cells, which sits at the intersection of many other functions that are crucial for maintaining cellular health. Identifying therapeutic targets to restore it and help clear damaging waste therefore holds promise as a treatment to protect motor neurons and slow MND progression.
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