FUS gene therapy
Dr Thomas Cunningham, Dr Rita Mejzini, Dr Loren Flynn and Professor Anthony Akkari, University College London and Murdoch University
Advancing Treatments Award
Accelerate new treatments
March 2025 - March 2028
£389,992.90
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.
Everyone has two copies of the FUS gene, but only one copy needs to carry a mutation for the person to be affected by MND. There are therapies targeting both copies of an individual’s FUS gene which are currently undergoing clinical trials, which reduce levels of mutant and healthy FUS. Dr Cunningham and his team are working on new therapies that target the FUS gene including an innovative approach to target only the mutated copy of FUS, stopping the production of toxic protein and allowing the unaffected copy to function normally.
Why is this research important
Although there are FUS therapies currently in clinical trials, this innovative approach could provide added benefits by reducing toxicities and providing longer treatment duration meaning less frequent administration may be needed. Lessons learnt by exploring innovative gene therapy approaches will also be valuable learnings for the wider field and inform the development of future treatments.
To learn more about gene therapies, check out our infographic.
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