Chief and Senior Investigator
Immune Deficiency Cellular Therapy Program, Center for Cancer Research, National Cancer Institute
Bethesda, Maryland, United States
Sung-Yun Pai MD is a physician-scientist trained in pediatric hematology, oncology, and stem cell transplantation, and has been a tenured Senior Investigator and Chief of the Immune Deficiency Cellular Therapy Program, Center for Cancer Research, National Cancer Institute, National Institutes of Health since 2020. Her research focuses on inborn errors of immunity (IEI) and its treatment with allogeneic hematopoietic stem cell transplantation and autologous gene therapy using viral vectors. Dr. Pai received her AB degree from Harvard University, MD degree from Harvard Medical School and completed training in pediatrics and pediatric hematology-oncology at Boston Children’s Hospital and Dana-Farber Cancer Institute. She was faculty there for 19 years, achieving the rank of Associate Professor and serving as the co-Director of the Gene Therapy Program. She led a seminal multi-institutional international trial of gene therapy for X-linked severe combined immunodeficiency (SCID-X1) using a modified self-inactivating gammaretroviral vector and reported in 2014 with European colleagues that this vector had equivalent efficacy to the parent vector with respect to immune reconstitution while avoiding insertional oncogenesis. She designed and is leading an open and enrolling trial for X-linked severe combined immunodeficiency (SCID-X1) using a lentiviral vector with low dose busulfan conditioning to enhance immune outcome, and published results of a trial of gene therapy for Wiskott-Aldrich syndrome. She has contributed to trials of gene therapy for chronic granulomatous disease and sickle cell anemia. She has published widely in the field of allogeneic transplantation for IEI, including SCID, Wiskott-Aldrich syndrome, DOCK8 deficiency, and others.
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Gene Therapy for Wiskott-aldrich Syndrome
Wednesday, June 19, 2024
1:40 PM – 2:05 PM PT
