Developing new FUS models to better understand how the protein becomes toxic in MND.

A photo of Juan

PhD Student: Juan Alcalde

Principal Investigator: Dr Marc-David Ruepp

Lead Institution: King's College London

MND Association Funding: £114,856*

Funding dates: October 2023 - September 2026

*Supported by the Heaton-Ellis Trust

About the project

MND is known to be associated with changes in a gene called Fused in Sarcoma (FUS). These mutations cause the FUS protein to be lost from the cell nucleus, where it is meant to reside, and to mislocate to the cell cytoplasm. In the cytoplasm of the cell, the FUS protein has a toxic function that damages and kills motor neurons. However, it is not clear what causes cytoplasmic FUS to be toxic. This project aims to develop new FUS-linked MND models to understand more about the interactions that FUS has with other proteins in the cell cytoplasm and how these may be involved in the disease. This could lead to help to determine how toxic FUS is responsible for damaging motor neurons and identify new targets for future therapy development for FUS-linked MND.

What could this mean for MND research?

Having better models to study FUS-linked MND will help to increase current understanding of how the FUS protein becomes toxic in the cytoplasm. This knowledge of the underlying biology of toxic FUS could lead to the discovery of new ways to counteract or reverse it’s toxicity, leading to the identification of new therapeutic targets for MND.

Project code: 911-792

Animal research

 

 

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