Abstract by Mary Vallecillo
Chemistry and Biochemistry
Pam Van Ry
rHsGal-1 Improves Membrane Repair Capacity in LGMD2B
Muscular dystrophies are a group of genetic diseases that involve gene mutations leading to decreased muscle strength, and membrane repair. Limb-Girdle Muscular Dystrophy 2B (LGMD2B) is a subtype of muscular dystrophy caused by mutations in the DYSF gene, which encodes for dysferlin protein. Functional dysferlin is thought to participate in resealing the membrane after natural injury events. Due to the lack of current treatment options, our goal is to assess the efficacy of recombinant human galectin-1 (rHsGal-1) as a novel therapeutic option for decreasing disease manifestation. Our data indicates that Gal-1 improves membrane repair capacity and helps in myogenesis, offering evidence that Gal-1 may favorably influence the outcome of LGMD2B. By using a laser injury ablation assay, we can determine if the treatment with rHsGal-1 will improve membrane repair capacity. These results suggest the potential of galectin-1 as a strong protein therapeutic for LGMD2B.