Sung-Yun Pai, MD

Department	Hematology/Oncology
Hospital Title
Academic Title	Assistant Professor in Pediatrics
Phone	617-919-2508
Fax	617-730-0934
Email	Sung-Yun Pai
Location	Karp Family Research Laboratories Room 08214 One Blackfan Circle Boston, MA 02115

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)

Key Publications

• McDonald DR, Massaad MJ, Johnston A, Keles S, Chatila T, Geha RS, Pai SY. Successful engraftment of donor marrow after allogeneic hematopoietic cell transplantation in autosomal-recessive hyper-IgE syndrome caused by dedicator of cytokinesis 8 deficiency. J Allergy Clin Immunol (2010) vol. 126 (6) pp. 1304-5.e3.

• Hale JE, Bonilla FA, Pai SY, Gerstel-Thompson JL, Notarangelo LD, Eaton RB, Comeau AM. Identification of an infant with severe combined immunodeficiency by newborn screening. J Allergy Clin Immunol (2010) vol. 126 (5) pp. 1073-4.

• Pai SY, Levy O, Jabara HH, Glickman JN, Stoler-Barak L, Sachs J, Nurko S, Orange JS, Geha RS. Allogeneic transplantation successfully corrects immune defects, but not susceptibility to colitis, in a patient with nuclear factor-kappaB essential modulator deficiency. J Allergy Clin Immunol (2008) vol. 122 (6) pp. 1113-1118.e1.

• Pai SY, Kang BY, Sabadini A , Parisini E, Truitt ML, Ho IC. Distinct Structural Requirements of GATA-3 in the Regulation of Thymocyte and Th2 Cell Differentiation. J. Immunol 2008 Jan 15; 180 (2): 1050-1059.

• Kim PJ, Pai SY, Brigl M, Besra GS, Gumperz J, Ho IC. GATA-3 Regulates the Development and Function of Invariant NKT Cells. J. Immunol. 2006 Nov 15; 177 (10): 6650-6659.

• Pai SY, DeMartiis D, Forino C, Cavagnini S, Lanfranchi A, Giliani S, Moratto D, Porta F, Imberti, L, Notarangelo LD, Mazzolari, E. Stem cell transplantation for the Wiskott-Aldrich syndrome: a single-center experience confirms efficacy of matched unrelated donor transplantation. Bone Marrow Transplant. 2006 Nov; 38 (10): 671-679.

• Pai SY, Truitt ML, Ho IC. GATA-3 deficiency abrogates the development and maintenance of Th2 cells. Proc Natl Acad Sci 2004; 101: 1993-1998.

• Pai SY, Truitt ML, Ting CN, Leiden JM, Glimcher LH, Ho IC. Critical roles for transcription factor GATA-3 in thymocyte development. Immunity 2003; 19: 863-875.

• Pai SY, Fruman DA, Leong T, Neuberg D, Rosano TG, McGarigle C, Antin JH, Bierer BE. Inhibition of calcineurin phosphatase activity in adult bone marrow transplant patients treated with cyclosporine A. Blood (1994) vol. 84 (11) pp. 3974-9