Personal Statement

I am the Director of the Inherited, Metabolic and Storage Disease Transplantation Program at the University of Minnesota. Our group is a world-leading center for the treatment of these disorders with cellular therapy, including hematopoietic stem cell transplantation and gene therapy. My training is in the field of Pediatrics, with subspecialty training in Hematology/Oncology and Blood and Marrow Transplantation. My clinical practice is now focused on the use of hematopoietic stem cell transplantation for non-malignant, inherited disorders, and better understanding the biology of these disorders. I have extensive experience in the design and implementation of clinical trials for inherited leukodystrophies, the mucopolysaccharidosies and other lysosomal disorders, as well as osteopetrosis. I have actively worked to build a team of professionals in various disciplines to provide optimal care for thesepopulations of patients. We areworking to establish methods to stratify risk and monitor patient outcomes using imaging, clinical parameters and biomarkers of disease.