Development of a personalized in-vitro model for dystrophic muscle endogenous repair for drug discovery
Duchenne muscular dystrophy (DMD) is a genetic disorder resulting in progressive muscle degeneration. Satellos Bioscience Inc is developing small molecule drugs that target and modulate DMD muscle stem cells to repair the dystrophic muscle. Despite the advantages of DMD animal models, they are not always predictive of human DMD phenotypes, or their response to drug treatments. In collaboration with the Gilbert lab, this project is focused on adapting an in-vitro model for muscle endogenous repair (MEndR) to include cells from dystrophic mice that can capture dystrophic muscle biology and recapitulate known biology of key published drugs in dystrophic mouse models. Next, to enable human testing of candidate drugs in a ‘personalized’ culture assay, we will incorporate induced pluripotent stem cells, derived from DMD patients, to create DMD MEndR, and then use this assay to evaluate Satellos’ lead therapeutic compounds.This project aims to develop a feasible model to bridge the gap between animal experiments and human clinical trials and enable Satellos to identify drug candidates most likely to ultimately succeed in clinical trials.