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Maria Grazia Roncarolo, MD, Prof. of Pediatrics, is the Chief of the Division of Pediatric Translational and Regenerative Medicine and Co-Director of the Institute for Stem Cell Biology and Regenerative Medicine.
She 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.
Dr. Roncarolo, a pediatric immunologist by training, spent her early career in Lyon, France, where she focused on severe inherited metabolic and immune diseases, including severe combined immunodeficiency (SCID), better known as “bubble boy disease.” Dr. Roncarolo 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, Dr. Roncarolo discovered a new class of T cells. These cells, called T regulatory type 1 cells, help maintain immune-system homeostasis by, among other things, preventing autoimmune diseases and helping the immune-system tolerate transplanted cells and organs. Recently, she completed the first clinical trial using T regulatory type 1 cells to prevent severe graft-versus-host disease in leukemia patients undergoing allogeneic hematopoietic stem cell transplantation.
Dr. Roncarolo worked for several years at DNAX Research Institute for Molecular and Cellular Biology in Palo Alto, where she contributed to the discovery of novel cytokines and she studied the role of cytokines in induction of tolerance and in promotion of stem cell growth and differentiation.
Dr. Roncarolo developed new gene-therapy approaches, which she pursued as director of the Telethon Institute for Cell and Gene Therapy at the San Raffaele Scientific Institute in Milan. She was the principal investigator leading the successful gene therapy trial for SCID patients lacking adenosine deaminase (ADA), a severe life-threatening disorder. The trial is now considered the gold standard for gene therapy in inherited immune diseases. Under her direction, the Institute has been seminal in showing the efficacy of gene therapy for other untreatable inherited metabolic diseases and primary immunodeficiencies.
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.
B-cell development and functions and therapeutic options in adenosine deaminase-deficient patients.
Brigida I, Sauer AV, Ferrua F, Giannelli S, Scaramuzza S, Pistoia V, Castiello MC, Barendregt BH, Cicalese MP, Casiraghi M, Brombin C, Puck J, Müller K, Notarangelo LD, Montin D, van Montfrans JM, Roncarolo MG, Traggiai E, van Dongen JJ, van der Burg M, Aiuti A.
J Allergy Clin Immunol. 2014 Mar;133(3):799-806.e10. - PubMed PMID: 24506932
Clinical features and follow-up in patients with 22q11.2 deletion syndrome.
Cancrini C, Puliafito P, Digilio MC, Soresina A, Martino S, Rondelli R, Consolini R, Ruga EM, Cardinale F, Finocchi A, Romiti ML, Martire B, Bacchetta R, Albano V, Carotti A, Specchia F, Montin D, Cirillo E, Cocchi G, Trizzino A, Bossi G, Milanesi O, Azzari C, Corsello G, Pignata C, Aiuti A, Pietrogrande MC, Marino B, Ugazio AG, Plebani A, Rossi P; Italian Network for Primary Immunodeficiencies.
J Pediatr. 2014 Jun;164(6):1475-80.e2. - PubMed PMID: 24657119
Forkhead box P3: the peacekeeper of the immune system.
Passerini L, Santoni de Sio FR, Roncarolo MG, Bacchetta R.
Int Rev Immunol. 2014 Mar;33(2):129-45. - PubMed PMID: 24354325
Immunological Outcome in Haploidentical-HSC Transplanted Patients Treated with IL-10-Anergized Donor T Cells.
Bacchetta R, Lucarelli B, Sartirana C, Gregori S, Lupo Stanghellini MT, Miqueu P, Tomiuk S, Hernandez-Fuentes M, Gianolini ME, Greco R, Bernardi M, Zappone E, Rossini S, Janssen U, Ambrosi A, Salomoni M, Peccatori J, Ciceri F, Roncarolo MG.
Front Immunol. 2014;5:16. - PubMed PMID: 24550909
Tr1 cells and the counter-regulation of immunity: natural mechanisms and therapeutic applications.
Roncarolo MG, Gregori S, Bacchetta R, Battaglia M.
Curr Top Microbiol Immunol. 2014;380:39-68. - PubMed PMID: 25004813