Discussion

ALS /MND is clinically, pathologically, genetically and prognostically heterogeneous, creating a challenge for therapy development. This presentation will describe the use of gene expression profiling in cellular and in vivo models of ALS, linked back to biosamples from ALS patients, to identify pathways of motor neuron injury which might be therapeutically manipulated. Approaches for neuroprotective therapy development using small molecule libraries and gene therapy tools will be described. Specific examples relating to up-regulation of the NRF2- anti-oxidant response element (ARE) pathway and siRNA approaches to reduce the expression of SOD1 using AAV9 viral vectors.