Developing a dual-action gene therapy for C9orf72-related MND.
Principal Investigator: Dr Christopher Webster
Lead Institution: University of Sheffield
MND Association Funding: £329,990
Funding dates: April 2025 - March 2028
About the project
This project targets the most common genetic cause of MND, a defect in the C9orf72 gene, which leads to the build-up of toxic proteins and motor neuron cell death. With no current cure and recent clinical trial failures for C9orf72-MND, this research aims to develop a new targeted therapy. The strategy involves using safe, engineered viruses to deliver a molecule called shRNA that removes the faulty C9orf72 gene while simultaneously supplying a healthy version. Early results on reducing faulty C9orf72 using viruses in mouse neurons show a reduction in the toxic proteins, supporting the potential of this dual-action approach as a future treatment.
What could this mean for MND research?
If successful, this therapy could offer a new way to treat C9orf72-related MND by directly targeting the genetic cause. The project will test the treatment’s ability to reduce toxic proteins, deliver a working gene copy, and assess safety and dosage in a mouse model, laying the groundwork for further research and potentially future clinical trials.
Project code: 2500-791
