Modeling SBMA: from understanding proteotoxicity to identifying therapeutics
Summary: They believe that the specific cell death is due to the accumulation of misfolded proteins (the androgen receptor) and the inability of cells to handle this accumulation. They propose to use a new model organism (a little worm called C. elegans – a very common and important model system in biology) to examine how different cell types handle the misfolded proteins and genetically look for other proteins that may help the cell get rid of the messed up proteins.