CDCM LEADERSHIP

Maria Grazia Roncarolo, MD

Director, Center for Definitive and Curative Medicine

Maria Grazia Roncarolo, MD is the George D. Smith Professor in Stem Cell and Regenerative Medicine, Professor of Pediatrics and of Medicine, director of the Center for Definitive and Curative Medicine, and co-director of the Institute for Stem Cell Biology and Regenerative Medicine.

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.

A pediatric immunologist by training, she earned her medical degree at the University of Turin, Italy. She 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 the "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.

Dr. Roncarolo established the Stanford Center for Definitive and Curative Medicine to cure patients with currently incurable diseases through the development of innovative stem cell-and gene-based therapies.

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Anthony E. Oro, MD, PhD

Co-Director, Center for Definitive and Curative Medicine

A developmental biologist and practicing dermatologist, Dr. Oro brings expertise in basic and translational mechanisms of epithelial regeneration and carcinogenesis. He helped make the connection between the hedgehog developmental pathways and human cancer, was a principal investigator for the first hedgehog pathway inhibitor for skin cancer, and has identified and developed lead compounds for novel drug resistance pathways. A discoverer of nuclear receptors like retinoic acid receptor, Dr. Oro studies chromatin dynamic changes in the developing genome that have led to the use of iPSCs for clinical manufacturing of corrected skin grafts for Epidermolysis bullosa.

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Matthew Porteus, MD, PhD

Co-Director, Center for Definitive and Curative Medicine

A pediatric hematologist/oncologist by training, Dr Porteus brings international leadership in basic and translational applications of genome editing. He has led the development of the field of genome editing and established the key approaches to using the CRISPR/Cas9 system in clinically relevant human somatic stem cells. The goal of his research is to develop safe and effective therapy for patients with monogenic diseases such as β-thalassemia, sickle cell disease, hemophilia, and severe combined immunodeficiency and for infectious diseases such as HIV using homologous recombination mediated genome editing. This platform can then be developed to treat genetic diseases of other organ systems and to enhance the potency and safety of cell based regenerative medicines.

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