Determine whether or not, and to what extent, the mutant androgen receptor (AR) in the muscle contributes to both muscle atrophy and motor neuron degeneration
Summary: Essentially this research will attempt to determine whether or not, and to what extent, the mutant androgen receptor (AR) in the muscle contributes to both muscle atrophy and motor neuron degeneration in Kennedy’s Disease (KD). They have produced a mouse model of KD in which they can remove the mutant AR gene just in the muscle cells (this is due to a genetic manipulation), leaving the mutant AR intact in the rest of the cells. This type of research will hopefully show us which cells and tissues are the most important for the timing of the appearance of KD symptoms (i.e., the onset) and/or the pace of their progression. With this knowledge, we will be able to develop our therapies for KD more effectively and target them to the appropriate tissues.
