IV Delivery of Ex Vivo Lentiviral Corrected CD34+ Bone Marrow Cells to Treat Systemic Disease in a Canine Model of Mucopolysaccharidosis VI
Awardee: Margret L. Casal
Institution: University of Pennsylvania
Grant Amount: $60,350.00
Funding Period: February 1, 2023 - January 31, 2024
Summary:
MPS VI causes severe skeletal disease and moderate systemic disease. Our canine model faithfully mimics the disorder in human patients with MPS VI. This projects examines the feasibility of harvesting stem cells from dogs affected with MPS VI, correcting the cells in tissue culture using viral vectors, and then returning the gene therapy corrected cells back to the affected neonatal dog. These experiments will show that this form of therapy is safe and effective, while not having the side effects of bone marrow transplantation (graft rejection, host versus graft disease, lifelong immunosuppression) or enzyme replacement therapy (repeated administration, expense, inability to correct bone disease).