FUS gene therapy

Summary

Mutations in a gene called Fused in Sarcoma (FUS) are known to play a role in the development and progression of MND and Frontotemporal Dementia (FTD). FUS-MND affects a small percentage of familial cases. It usually has earlier onset and faster progression. The accumulation of mutant FUS protein in motor neurons is toxic. 

Dr Lee and his team have developed an adeno-associated virus (AAV) gene therapy to target and reduce the levels of the harmful FUS protein. This project aims to test the efficacy of the therapy when delivered directly to the brain and spinal cord of mice.

Why is this research important

If successful, this proof-of-concept research could pave the way for further development of a treatment for FUS-related MND. Compared to antisense oligonucleotide therapies targeting FUS which are currently in clinical trials, an AAV gene therapy would only need to be administered once making the treatment much less burdensome for people living with MND.

*This project is co-funded with MND Association with a total value of £99,933.25.

To learn more about gene therapies, check out our infographic.