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.
Related Content
-
Increased acute care utilization in a prospective cohort of adults with sickle cell diseaseThe ESCAPED (Examining Sickle Cell Acute...
-
Caries prevalence and impact on oral health-related quality of life in children with sickle cell disease: cross-sect...BACKGROUND: Children with sickle cell di...
-
Robert Molokie, MDDr. Robert Molokie is a hematologist and...
-
Guy’s and St Thomas’ NHS Foundation TrustOur haemoglobinopathies service sees chi...
-
Steven Hardy, PhDSteven Hardy, Ph.D. is the Director of P...
-
WHATZ DA COUNTWDC on SCT has dedicated his Organizatio...
-
Healthy red blood cells owe their shape to muscle-like structuresRed blood cells are on a wild ride. As t...