General Grant

Bones, joints and cardiovascular tissues are among the most difficult to treat with the standard therapies available today for MPS I. Disease affecting these tissues also causes the most disability in our patients, including poor quality of life and risk of early death. Inflammation is a mechanism that seems to explain much of the bone and joint disease in animal models of mucopolysaccharidoses. In this project, we are studying a specific inflammatory pathway in vascular disease, which also occurs in MPS patients. This alternative inflammatory pathway is hypothesized to be stimulated by angiotensin II, a molecule that plays a role in

atherosclerosis and coronary artery disease. We plan to test the importance of this pathway by studying vascular smooth muscle cells from MPS I mice and we plan to test a treatment for this type of inflammation using an angiotensin receptor blocker in MPS I mice.

–¬†Moin Vera, MD, PhD

Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center Torrance, CA