CDCM LEADERSHIP

Maria Grazia Roncarolo, MD

Director, Center for Definitive and Curative Medicine

A pediatric immunologist by training, Dr Roncarolo is a world expert in genetic diseases, immune tolerance and gene therapy. She discovered, characterized and brought to the clinic a new subset of human T cells (Tr1) with immune regulatory and suppressor functions. She designed and executed gene therapy trials for primary immunodeficiencies and other genetic diseases. She was the principal investigator for the first successful stem cell gene therapy trial for severe combined immunodeficiency (SCID) due to adenosine deaminase deficiency, better known as the "bubble boy disease”, which received European Commission approval to market under the name of Strimvelis.

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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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