Continued development and testing of a potential one-time gene therapy for people living with FUS-MND.
Principal Investigator: Dr Younbok Lee
Lead Institution: King's College London
MND Association Funding: £49,966.62*
Funding dates: February 2025 - November 2025
*This project is co-funded with My Name’5 Doddie Foundation with a total value of £99,933.25.
About the project
In some cases of MND, people may have changes in the FUS gene. This causes 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.
Dr Lee and colleagues have developed a potential gene therapy that aims to reduce the activity of the faulty FUS gene. The therapy uses 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. This current project aims to continue the development of this therapy by testing the treatment in mice that have been genetically modified to mimic the disease.
What could this mean for MND research?
If successful, this project could lead to the development of a new one-time therapy for people living with FUS-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.
Project code: 2501-791
