Dr. Maria Grazia Roncarolo of Stanford University is a pediatric immunologist, the Chief of the Division of Pediatric Translational and Regenerative Medicine, and Co-Director of the Institute for Stem Cell Biology and Regenerative Medicine. An expert in stem cells and gene therapy, she is the director of recently announced Stanford Center for Definitive and Curative Medicine, which will work to tun lab discoveries into therapies to aid the millions of people with genetic diseases.
Roncarolo spent her early career in Lyon, France where she focused on sever inherited metabolic and immune diseases. She was a key member of the team that carried out the first stem cell transplants given before birth to treat these genetic diseases.
While studying inherited immune diseases, Roncarolo discovered a new class of T cells (T regulatory type 1 cells). These cells help maintain immune-sytstem homeostasis by preventing autoimmune diseases, as well as helping the immune system tolerate transplanted cells and organs. Recently, she completed her first clinical trial involving T regulatory type 1 cells to prevent severe graft-versus-host disease in leukemia patients undergoing allogeneic hematopoietic stem cell transplantation.
Ronarolo spent several years at DNAX Research Institute for Molecule and Cellular Biology in Palo Alto. In addition, she developed new gene-therapy approaches as director of the Telethon Institute for Cell and Gene Therapy at San Raffaele Scientific Institute in Milan.
“Dr. Roncarolo’s goal at Stanford is to build the teams and infrastructures to fast track stem cell and gene therapy to the clinic and to bring basic-science discoveries to patients. In addition, her laboratory continues to work on T regulatory cell-based treatments to induce tolerance after transplantation of allogeneic tissue stem cells. She recently published in Nature Medicine new biomarkers for T regulatory type 1 cells, which will be used to purify the cells and to track them in patients. She is also investigating genetic chronic inflammatory and autoimmune diseases due to impairment in T regulatory cell functions.”