Abstract by Mary Vallcillo
Chemistry and Biochemistry
Pam Van Ry
Recombinant Galectin-1 Protein Therapy in Limb Girdle Muscular Dystrophy Type 2B
Recombinant Galectin-1 (Gal-1) protein therapy was effective in Duchenne muscular dystrophy which has some pathophysiological end point measures overlapping with Limb Girdle Muscular Dystrophy Type 2B (LGMD2B). Lack of dysferlin protein leads to diminished plasma membrane repair in LGMD2b. Gal-1 is maximally expressed during myoblast fusion and evidence show overexpression of Gal-1 results in increased fusion into mature muscle cells resulting in increased repair. In LGMD2B, muscle deterioration is accelerated by an overactive inflammatory response. We hypothesize that Gal-1 will improve membrane repair through immune modulation thus stabilizing muscle integrity/strength and increasing myogenesis in models of LGMD2B. Human recombinant Gal-1 (rHsGal-1) was produced and verified through western blot analysis and BCA Protein assay. We plan to treat LGMD2B muscle cells with rHsGal-1. Improved myogenesis will be evaluated to determine optimal dose of Gal-1. We will also record cytokines involved in the pro-inflammatory response.