Advances in Bioanalytics and Biomarkers
Target and Mechanism Deconvolution After Phenotypic Screens
Motor neuron diseases, as a class, are becoming increasingly well-known and understood. However, effective therapeutics that preserve motor neurons are still lacking. Obviously, motor neuron dysfunction is reflected, ultimately, in skeletal muscle weakness and deterioration. Surprisingly, the involvement of muscle cells themselves in diseases ranging from Spinal Muscular Atrophy, a childhood developmental disorder, to sarcopenia – muscle weakness with aging – is much less understood. I will describe two different projects – one directed at promoting the survival of healthy motor neurons, the other at improving muscle health. Both projects started with disease-relevant cell-based screens and culminated in the discovery of new therapeutic targets.