6-month treatment with galectin-1 improves LGMD2B disease pathology in dysferlin-deficient mice

Abstract

Limb-girdle muscular dystrophy type 2B (LGMD2B) is a devastating autosomal recessive disease for which there are no current FDA-approved treatments. Our previous research shows that short-term recombinant human Galectin-1 (rHsGal-1) treatment is effective in improving muscle repair and decreasing fat deposition in the BLA/J mouse model. Our current objective is to determine the efficacy of a long-term (6-month) weekly treatment with rHsGal-1 in improving disease pathology in BLA/J mice. Mice treated weekly with intraperitoneal injections of 2.7 mg/kg rHsGal-1 show significant improvements in the time it takes to cross a 10 mm wide balance beam in comparison with saline-injected mice. Treated mice also show less decline over time in how often they rear on their hind legs while exploring a new environment, which suggests improved pathology of the psoas and gluteus muscles, which are especially affected in the BLA/J model. Oil Red O staining of the psoas muscles shows decreased lipid deposition in the muscle of rHsGal-1 treated mice. Other histological stains show improved pathology in various affected muscles. We conclude that weekly treatment with rHsGal-1 is a promising therapeutic for individuals with LGMD2B. This research is funded by the Jain Foundation This is the full abstract presented at the American Physiology Summit 2023 meeting and is only available in HTML format. There are no additional versions or additional content available for this abstract. Physiology was not involved in the peer review process.