Galectin-1: A potential protein therapy for Limb Girdle Muscular Dystrophy type 2B

Pamela Van Ry, PhD

Brigham Young University, Provo, Utah

Pam Van Ry currently works at the Department of Chemistry and Biochemistry, Brigham Young University in Provo, Utah. Pam does research in Neuroscience, Physiology and Molecular Biology. Their current project is 'Protein therapy in LGMD2B: defining protein homeostasis'. The Van Ry Lab also researches causes and therapies for Pulmonary Fibrosis.

Research Projects

Our overall objective is to develop a novel therapeutic intervention that increases muscle membrane repair and modulates inflammation away from a chronic response in LGMD2B to improve overall patient outcomes.

Our scientific rationale is built on prior reports that galectin-1 can increase muscle stability and decrease inflammation which will lead to improved muscle membrane repair. Our preliminary data provides the first evidence demonstrating the ability of Gal-1 to favorably improve LGMD2B disease progression by increasing membrane repair capacity after laser injury, increasing muscle formation and decreasing inflammatory markers. Our proposal will define Galectin-1 as a favorable suppressor of LGMD2B disease pathogenesis and define the mechanism through which these changes are accomplished. Since there are different biochemical forms of Galectin-1 (each known to have unique functions), it is crucial to define the biochemical requirements responsible for therapeutic benefit in LGMD2B. Chronic muscle inflammation is a significant disease characteristic in LGMD2B.1, 2 We will measure the changes in inflammation (macrophage polarization) with Galectin-1 treatment. This proposal uses models of LGMD2B to test the hypothesis that Gal-1 treatment decreases disease pathology by increasing membrane repair capacity and shifting immune homeostasis away from chronic inflammation. Successful completion of this project will define key biochemical characteristics of Galectin-1 necessary for increasing membrane repair and the impact of Galectin-1 on inflammation in LGMD2B.  In doing so, these studies will provide new insight into Galectin-1 as a therapeutic for LGMD2B.