公开(公告)号：US20210308218A1

公开(公告)日：2021-10-07

申请号：US17224493

申请日：2021-04-07

Applicant: Brigham Young University

Inventor： Pam Van Ry ,  Mary Vallecillo

IPC: A61K38/17 ,  A61K9/00

Abstract: Limb-girdle muscular dystrophy type 2B (LGMD2B) is caused by mutations in the dysferlin gene, resulting in non-functional dysferlin, a key protein found in muscle membrane. Treatment options available for patients are chiefly palliative in nature and focus on maintaining ambulation. A method of treating LGMD2B is disclosed herein. The method includes administering to a patient a suitable amount of a galectin protein or fragment thereof. Treatment with a recombinant galectin promoted myogenic maturation as indicated through improvements in size, myotube alignment, myoblast migration, and membrane repair capacity in dysferlin-deficient myotubes, explant myofibers and mice.