Research Overview

The Pai laboratory focuses on the study of disorders of human T and B cell development and function, and treatment of these disorders by allogeneic hematopoietic cell transplantation and gene therapy. Her previous work include cellular studies of the mechanism of action of immunosuppressive agents such as cyclosporine, tacrolimus and rapamycin, and key contributions on the role of GATA-3 in transcriptional regulation of CD4 T cell development and T helper cell differentiation.

Current projects in the laboratory include study of patients with immunodeficiency, particularly with severe combined immunodeficiency (SCID) and its variants and Wiskott-Aldrich syndrome, both pre and post-cellular therapy. These studies include in vitro differentiation of T progenitors from hematopoietic stem cells and in vivo development in humanized mouse models. The Pai laboratory also studies immune reconstitution and function in patients undergoing gene therapy for X-linked SCID and Wiskott-Aldrich syndrome.

She is also involved in the development and implementation of universal newborn screening for SCID in Massachusetts, and is a member of a North American consortium for study of treatment of immunodeficiency, the Primary Immune Deficiency Treatment Consortium (PIDTC).

About Sung-Yun Pai

Dr. Pai received her MD degree with honors from Harvard Medical School in 1994. She trained in pediatrics at Children's Hospital in Boston from 1994 to 1997 and in pediatric hematology-oncology at Dana-Farber Cancer Institute/Boston Children's Hospital from 1998 to 2001.

She has been on staff at Children's Hospital and Dana-Farber Cancer Institute since 2001. She evaluates patients with primary immunodeficiency for curative hematopoietic stem cell transplantation.

Clinical trial links:

Gene Transfer for Severe Combined Immunodeficiency, X-linked (SCID-X1) Using a Self-inactivating (SIN) Gammaretroviral Vector, Site Co-investigator (NCT01129544)

Pilot and Feasibility Study of Hematopoietic Stem Cell Gene Transfer for the Wiskott-Aldrich Syndrome, Principal Investigator (NCT01410825)

Natural History Study of SCID Disorders, Site Principal Investigator (NCT01186913)

Patients Treated for SCID (1968-2010), Site Principal Investigator (NCT01346150)

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  • T Cell Development and Function

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