Restoring Immune tolerance through Cell and Gene Therapy

Immune mechanisms and curative treatments

Lab overview


Dr. Roncarolo's goal at Stanford is to build the teams and infrastructure to move stem cell and gene therapy to the clinic quickly and to translate basic science discoveries into patient treatments. In addition, her laboratory continues to work on T regulatory cell-based treatments to induce immunological tolerance after transplantation of donor tissue stem cells. In Nature Medicine, Dr. Roncarolo recently published her discovery of new biomarkers for T regulatory type 1 cells, which will be used to purify the cells and to track them in patients. She also is investigating genetic chronic inflammatory and autoimmune diseases that occur due to impairment in T regulatory cell functions.


Research Interests

  • Immunotolerance: Mechanisms underlying T-cell tolerance, induction of T-cell anergy and regulatory T cells; Immunomodulation: mAbs, proteins and low molecular weight compounds which can modulate T-cell activation; Primary immunodeficiencies: Characterization of molecular and immunological defects;
  • Gene therapy: Gene transduction of hematopoietic cells for gene therapy in primary immunodeficiencies and metabolic diseases;
  • Hematopoiesis: Mechanisms underlying growth and differentiation of hematopoietic stem cells;
  • Transplantation: Immune reconstitution and T-cell tolerance after allogenic stem cell transplantation;
  • Cytokines/Cytokine receptors: Role in regulation of immune and inflammatory responses.


Clinical Interests

  • Primary Immunodeficiencies
  • Monogenic Autoimmune Disorders
  • Allogenic Bone Marrow Transplantation 
  • Gene Therapy Clinical Trials 
  • Cell Therapy Clinical Trials
  • Clinical Trials in Autoimmune Diseases and Organ Transplantation
  • Clinical Trials in Hemoglobinopathies

Maria Grazia-Roncarolo, MD: Stanford Childx Conference

Maria Grazia Roncarolo, MD

Maria Grazia Roncarolo, MD is the co-director of the Institute for Stem Cell Biology and Regenerative Medicine, the George D. Smith Professor in Stem Cell and Regenerative Medicine, Professor of Pediatrics and of Medicine (blood and marrow transplantation), chief of the Division of Pediatric Stem Cell Transplantation and Regenerative Medicine, and co-director of the Bass Center for Childhood Cancer and Blood Diseases. 


Dr. Roncarolo leads efforts to translate scientific discoveries in genetic diseases and regenerative medicine into novel patient therapies, including treatments based on stem cells and gene therapy.

Stanford Resources

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