Developing a new potential gene therapy for people living with FUS-MND.
Principal Investigator: Dr Younbok Lee
Lead Institution: King's College London
MND Association Funding: £250,000*
Funding dates: October 2022 -September 2024
*Funded in partnership with LifeArc and supported by the Heaton Ellis Trust.
About the project
In some cases of MND, people may have changes in the FUS gene which cause a faulty FUS protein to be made that builds-up in the wrong area of the neuron. This disturbs the production of other proteins that are essential to motor neuron health and survival, leading to motor neuron damage. Previous research has shown that reducing the activity of the altered FUS gene leads to less of the faulty FUS protein being made and may be able to prevent further damage to motor neurons. This project focuses on developing a potential gene therapy that aims to reduce the activity of the faulty FUS gene. The therapy will use a compound called an Antisense oligonucleotide (ASO) which targets the faulty FUS gene and is designed to stop the faulty protein from being made. This potential new ASO therapy will be designed to work long-term with only one treatment needed and, if successful, could help prevent further motor neuron loss.
What could this mean for MND research?
If successful, this project could lead to the development of a new potential therapy for people living with MND. This project will focus on the pre-clinical work needed to see if the potential therapy will have any benefit and if it is safe. If the therapy is found to show benefit and is safe it has the potential to move onto human clinical trials.
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Project code: 993-797
