Unveiling regenerative and metabolic features of SBMA muscle cells to identify new therapeutic targets.
Skeletal muscle cells have a primary role in the SBMA pathogenesis. The aim of this project is to clarify how the elongated androgen receptor induces muscle fiber dysfunctions. To achieve this goal, we will take advantage of induced pluripotent stem cells differentiated to skeletal muscle cells, and of skeletal muscle of an SBMA mouse model at different disease stages. We will focus on two different processes: the metabolism and the regenerative ability of SBMA affected muscle cells. Explaining the mechanisms of toxicity of the elongated androgen receptor in muscle will permit us to target therapeutics to key pathological stages, and to identify useful biomarkers for disease diagnosis and the reading of clinical trial results.
