SRSF1 gene therapy
Professor Guillaume Hautbergue,Professor Dame Pamela Shaw and Dr Monika Myszczynska, Sheffield Institute for Translational Neuroscience (SITraN), The University of Sheffield
Advancing Treatments Award
Accelerate new treatments
March 2025 - March 2028
£293,186.00
Summary
Professor Hautbergue and his team previously developed a gene therapy targeting a RNA-binding protein called SRSF1. This showed efficacy in models of C9Orf72 MND, the most commonly mutated gene in familial MND. The therapy is moving towards clinical trials for people with C9Orf72.
In the majority of people with sporadic MND, a protein called TDP-43 forms toxic clumps. Interestingly Prof Hautbergue and his team showed that targeting SRSF1 reduced these TDP-43 toxic clumps and promoted survival of motor neuron models of sporadic MND. This project will evaluate the potential of targeting SRSF1 in sporadic MND and understand the mechanisms behind its effects on TDP-43.
Why this research is important
This project could open up the door to a new treatment option for people with sporadic MND. Because the gene therapy is already being progressed for C9Orf72 MND, it could mean it would be even faster to move the therapy into a wider MND population if the results look promising.
To learn more about TDP-43 take a look at our TDP-43 infographic and to understand more about gene therapies, check out our gene therapy infographic.
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