I am an international expert in cell and gene therapy for patients with inherited metabolic and lysosomal storage disorders, including adrenoleukodystrophy (ALD), metachromatic leukodystrophy (MLD), mucopolysaccharidosis type I (MPS I), and osteopetrosis (OP). As a recognized key opinion leader in these areas, I have over a decade of experience consulting on various aspects of rare diseases and clinical research to help advance therapeutic options and improve patient outcomes.At the University of Minnesota, I serve as a co-investigator on all protocols to treat ALD, MLD, MPS I, and OP with hematopoietic stem cell transplantation. My research focuses on important clinical questions related to the biology and treatment of these rare diseases. I am particularly interested in identifying biomarkers that can predict neurocognitive outcomes, specifically IQ. I oversee one of the world's largest biorepositories for these rare diseases. I enjoy partnering with colleagues and industry to develop safer, more effective therapies that can improve outcomes and save lives.I have published extensively, with over 150 articles in peer-reviewed journals such as Blood, Biology of Blood and Marrow Transplantation, Stem Cells, Nature Reviews Clinical Oncology, and Blood Advances. Additionally, I have presented more than 200 abstracts and lectures at national and international meetings, sharing insights and advances in the field of rare disease treatment and research.