Unravelling TDP-43 autoregulation – routes to therapy in MND

£115,520 was donated by 22 branches and groups towards this research project which is using cutting edge approaches to test thousands of different potential gene therapies on several MND disease models, with the aim of identifying ways of reducing TDP-43 to normal levels. Because of the central role that TDP-43 plays in MND, this project could pave the way for new treatment development.

"This project is of high importance, being a new and innovative attempt to understand how TDP-43 pathology is triggered in disease and to identify ways to prevent it."

Scientific peer reviewer

Project update: So far, the research team has engineered and tested three ‘reporter’ molecules that can indicate the levels of TDP-43 in human cells without interfering with any cell functions. Next, the reporters will be used to screen across almost the entire human genome to identify ways to correct the TDP-43 imbalance.