Google
Trusted Resources: People & Places
Healthcare providers, researchers, and advocates
Maria Grazia Roncarolo, MD
Healthcare Provider Professor of Pediatrics
Lorry I. Lokey Stem Cell Research Building
265 Campus Drive West
Room G3021A
Stanford, California, United States
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 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 who lack an enzyme critical to DNA synthesis, which is a severe life-threatening disorder. That trial is now considered the gold standard for gene therapy in inherited immune diseases. Under her direction, the San Raffaele Scientific Institute has been seminal in showing the efficacy of gene therapy for otherwise untreatable inherited metabolic diseases and primary immunodeficiencies. Her 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.
+myBinderRelated Content
-
Health Care Utilization Trajectories and Associated Factors for Transitioning Adolescents/Young Adults with Sickle C...Limited research had explored health car...
-
How Sickle Cell Anaemia Impacts Families and How We Can Helphttps://www.youtube.com/watch?v=5GUiuXQ9...
-
John T. Horan MD, MPHJohn T. Horan, MD, MPH, is Associate Pro...
-
Adult Sickle Cell Clinic at UAMSThe University of Arkansas for Medical S...
-
Trends in Sickle Cell Disease-related Priapism in U.S. Children’s HospitalsOBJECTIVE: To define rates of priapism ...
-
Colorado Sickle Cell Research and Treatment CenterColorado Sickle Cell Treatment and Resea...
-
Lamia Barakat, PhDLamia P. Barakat, PhD, is the Director o...
