Developing novel disease-specific gene therapy vectors for MND

Impact Grant
This project proposes to develop gene delivery vectors that selectively target cells affected by majority of MND cases affected by the protein TDP-43. This project will lead to new gene delivery vector discovery that selectively target and treat cells affected by MND. This will lead to improved targeting accuracy but also minimise off-target effects, aligning perfectly with the emphasis of the grant scheme on therapeutic precision and safety of gene therapy.