Primary Menu

Education, Events, Publication

Funding & Recognition

rHsGal-1: A Novel Protein Therapy for LGMD2B

Year: 2023

Presenter Name: Parker Nelson

Limb-girdle muscular dystrophy type 2b (LGMD2B) is an autosomal recessive disease caused by a mutation in dysf gene, the gene responsible for the key membrane repair protein called dysferlin. Unlike similar degenerative diseases, there is no viable treatment for LGMD2B. In the Van Ry Lab, our research is focused on understanding whether recombinant human Galectin-1 (rHsGal-1) could serve as a viable therapeutic option based on previous studies published evidencing its efficacy in treating Duchesne Muscular Dystrophy. A six-month study was conducted in dysferlin-deficient (BLA/J) mice under which time various assays and experiments were conducted, including monthly blood serum analyses and functional movements tests. Further tests were conducted to elucidate the effects of rHsGal-1 in the murine model, such as a laser injury assay of myofibers and histological examinations to study muscle morphology. Results from tests conducted demonstrated a statistically significant difference between treated and non-treated mice. Membrane repair, muscle morphology, and overall muscular health were improved in treated mice over a six-month period. Continued understanding of the role rHsGal-1 plays in membrane repair will help the thousands of individuals affected by LGMD2B worldwide.
University / Institution: Brigham Young University
Type: Oral
Format: In Person
SESSION B (10:45AM-12:15PM)
Area of Research: Science & Technology
Faculty Mentor: Pam Van Ry
Location: Union Building, PANORAMA EAST (11:05am)