Investigating the role of TBK1 in MND and whether targeting its activity could be a good strategy for future therapies.  

Valeria Gerbino

 

Principal Investigator: Dr Valeria Gerbino

Lead Institution: Fondazione Santa Lucia

MND Association Funding: £185,000*

Funding dates:November 2022 - October 2025

*Supported by Tony Hopper's friends and family

About the project

The progression of MND is thought to be influenced by cells in the nervous system called microglia. Previous research has shown that a gene called TBK1, which is involved in the immune response, becomes overactive in microglia in MND and this may contribute to disease progression. This project aims use cell and mouse models of MND to investigate whether reducing the activity of TBK1 in microglia could lead help to slow disease progression. The researchers will also test gene therapy approaches in these models to determine if targeting the activity of TBK1 could be a good therapeutic target for future therapy development.

What could this mean for MND research?

This project is looking to further understand the underlying biology happening in MND. Understanding more about TBK1 and the biological pathways can allow researchers to investigate if there are ways of correcting the damage done. This work will help to find new targets within the body and develop new therapies for these targets that could be beneficial in slowing disease progression.

Project code: 883-791

 

Animal research