Collagen-VI Supplementation by Cell Transplantation Improves Muscle Regeneration in Ullrich Congenital Muscular Dystrophy Model Mice

Type 6 collagen (COL6) is produced by mesenchymal stromal cells (MSC) in skeletal muscle, and its gene mutation the cause of Ullrich congenital muscular dystrophy (UCMD). To test whether COL6 supplementation has a therapeutic effect for UCMD, we conducted vivo vitro experiments using healthy induced pluripotent stem cell (iPSC)-derived MSCs (iMSCs) COL6-deficient iMSCs (KO-iMSCs). The transplantation these or primary into muscle immunodeficient UCMD model mice demonstrated that regeneration maturation were promoted only region supplemented with COL6. In addition, co-culture satellite derived from (Col6a1KO-MuSCs) showed but not KO-iMSCs could improve proliferation, differentiation, Col6a1KO-MuSCs. These findings indicate improves will help elucidate pathology establishment effective treatments.