Project One: Assessing and modulating the role of inflammation and fibrosis in the dystrophic process



H. Lee Sweeney, PhD, University of Florida

Melissa Spencer, PhD, UCLA

From its inception, the main goal of this Wellstone MDCRC has been to promote the translation of promising emerging therapeutic targets into clinical trials for muscular dystrophy patients. As such, there has been one project devoted to the discovery of new therapeutic targets, one project examining possible therapeutics and their impact on disease models of muscular dystrophy, and one project devoted to building tools necessary for human clinical trials in the muscular dystrophies.

Aim 1: Delineation of the cells that contribute to fibrosis and fat

Aim 2: Impact of genetic modifiers of inflammation and fibrosis on the cells involved in fibrosis

Aim 3: Modulation of fibrosis using therapeutics to decrease inflammatory and fibrotic responses and TGF-b1 signaling on the DBA background