Nucleoredoxin (NRX), a novel gene therapy target against TDP43 multifaceted pathogenic mechanisms.

Specific molecules have the ability to regulate oxygen levels in the brain. However, during MND, oxygen rich species increase, which induces neurodegeneration. We have identified a novel molecule, nucleoredoxin (NRX), that is a global regulator of oxygen species in cells and is protective in MND. Gene therapy approaches are showing great promise for neurodegenerative disorders, and using a completely new strategy, we will examine if delivering NRX is protective against disease onset/progression in mice that develop MND. This study will therefore examine if delivering NRX by this strategy has potential as a new treatment for MND in the future.