Developing a cutting-edge gene therapy that seeks to correct mistakes in the UNC13A gene in neurons.
Principal Investigator: Prof Pietro Fratta
Lead Institution: University College London
MND Association Funding: £164,291*
Funding dates: January 2023 - December 2025
* As part of the MND Translational Research Fund
About the project
Previous research has shown that a protein called TDP-43 is faulty in around 97% of people with MND. This faulty TDP-43 function has been found to cause a mistake to occur in the genetic instructions for another protein called UNC13A. The UNC13A protein plays an important role in allowing neurons to communicate with each other using chemical signals and, if the protein is not working as it should this could contribute to the neuron damage that occurs in MND. It has been suggested that the mistake in the instructions for the UNC13A protein might be corrected using small DNA-like molecules. This project aims to test small DNA-like molecules in cell and mice models of MND to see how effective they are at correcting the mistake and identify one that might be the most promising for use as a potential gene therapy for MND.
What could this mean for MND research?
This project aims to further test the small DNA-like molecules to identify which one might be the most promising for use as a potential gene therapy for MND. This project will also assess whether these molecules are safe to use and what dose would be required to achieve the desired effects. The most promising one could be further developed and tested in clinical trials for use as a gene therapy for people living with MND.
Resources
Project code: 995-797
