Uncovering a Role for Endothelial Cells in Duchenne Muscular Dystrophy

Dr. Rengarajan's research focuses on Duchenne muscular dystrophy (DMD), one of the most common and severe genetic diseases of childhood. Her goals are to better understand how DMD affects different tissues in the body and how current treatments affect the disease process. Eventually, she hopes this research will identify new targets for future treatments that improve the lives of individuals with DMD and stop progression of this disease.

During this research they will use muscle biopsies from individuals with and without DMD to look at the gene sequences of different cells in muscle. Dr. Rengarajan's mentors, Drs. Stan Nelson and Carrie Miceli, have an extensive and high-quality biobank with over 200 muscle biopsies, which allows her team to ask novel questions about this disease and delve into the effect treatments like gene therapy have on the disease's course.

Dr. Rengarajan and her team are focusing on the role of endothelial cells, which play a critical role in muscle health since they regulate muscle repair and regeneration and control how immune cells reach muscle. They are implicated in adverse effects of gene therapy.