Abstract by Spencer Hayes
Chemistry and Biochemistry
Pam Van Ry
Galectin-1 Protein Therapy Increases the Membrane Repair Capacity in Cell Models of LGMD2B
Limb-girdle muscular dystrophy type 2B (LGMD2B) is a severe, incurable, debilitating disease where the muscles of major skeletal muscle groups are weakened and atrophied. LGMD2B is caused by mutations in the DYSF gene which encodes for the dysferlin protein. An absence or mutation of dysferlin consequently leads to defective muscle repair and subsequent degeneration of muscles due to its vital role in the cellular membrane repair process. Our lab currently focuses on assessing the therapeutic potential of varying forms of Galectin-1 (Gal-1) in LGMD2B, one specifically being membrane repair capacity. Gal-1 is a small, naturally occurring protein expressed in all parts of the cell within various tissues in a monomeric, dimeric and multimeric form. We show through laser ablation assays that a recombinant form of Gal-1 (rHsGal-1) has more favorable repair potential in membrane repair than a fixed monomeric form of Gal-1, thus providing evidence for a probable therapy for LGMD2B.