Role of survival motor neuron deficiency in motor neuron disease

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The aim of this proposal is to understand the earliest and central pathological events in motor neuron disease (MND). This is important for drug development because therapies that target the early disease stages are likely to be effective. Our previous studies suggest that a protein essential for nerve cells, survival motor neuron, is lacking in MND. We will therefore examine how early this deficiency starts in disease and whether its replacement is beneficial. These outcomes may aid future studies to design new treatments for MND.