Abstract by Mary Vallecillo
Chemistry and Biochemistry
Pam Van Ry
Galectin-1 Protein Therapy Decreases Disease Pathology in Cell Models of Limb Girdle Muscular Dystrophy 2B
Limb Girdle Muscular Dystrophy 2B (LGMD2B) is an autosomal disease caused by mutations in the DYSF gene which is responsible for codifying functional dysferlin protein. Major characteristics of LGMD2B are increased inflammatory infiltrate and defective membrane repair in muscles cells. The lack of treatment options for LGMD2B patients requires increased research to slow disease progression. Galectin-1 is a carbohydrate recognition protein capable of stabilizing the sarcolemma. Here, we produced and used recombinant human Galectin-1 to treat dysferlin deficient cells. We show that Galectin-1 treatment improves fusion index, membrane repair capacity, increases LGALS1 mRNA levels, myogenic markers and decreases autophagy in dysferlin deficient myotubes. These results provide novel evidence that Galectin-1 is a potential therapeutic that favorably improves muscle function in LGMD2B.