Investigator: Darko Bosnakovski DVM PhD
Category: Research - Translational
Accumulation of the fibrous and fat tissue between atrophic myofibers is the hallmark of FSHD pathohistology. Fibroadipogenic progenitors are resident cells in the muscle that are the main contributors in the synthesis and deposition of the excessive extracellular matrix in the muscle. Fibrosis that usually progresses to scar formation negatively affects muscle function. We generated a mouse model for FSHD that closely resembles FSHD pathology. Our animal model allows us to study the mechanism of the diseases and to test different therapies. Currently, there is no specific therapy for FSHD and the patients are in great need of supportive therapy that will moderate the symptoms and improve their quality of life. Managing and reversing fibrosis is one key approach that can improve muscle function in FSHD patients. In this proposal, we plan to test the effectiveness of different FDA-approved antifibrotic drugs for treating muscle fibrosis in the FSHD animal model. We hope that our study will be an initiator for further research that will develop antifibrotic therapy that is applicable for FSHD.
NOTE: NCE for 3.5 months approved. 14-May-2022 -> 31-Aug-2022.