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Denise E. Sabatino, PhD
Denise E. Sabatino

The research in the Sabatino Laboratory is focused on hemophilia, an inherited bleeding disorder. The interests of the laboratory include the study of variants of coagulation factor VIII to understand the biochemical properties of these proteins and to identify novel variants with enhanced function, and the development of gene-based therapeutic approaches for treating hemophilia.



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Dr. Sabatino is a research assistant professor of Pediatrics at the Perelman School of Medicine at the University of Pennsylvania and Children's Hospital of Philadelphia. Dr. Sabatino completed her undergraduate studies at The Ohio State University and earned her PhD in Genetics at The George Washington University/National Institutes of Health in the laboratory of Dr. David Bodine at National Human Genome Research Institute.

During her postdoctoral fellowship with Dr. Katherine High at Children's Hospital, Dr. Sabatino investigated gene transfer for hemophilia B using adeno-associated viral (AAV) vectors. She joined the faculty at the University of Pennsylvania School of Medicine in 2008 in the Department of Genetics. In 2010 she joined the Department of Pediatrics and became a member of the Division of Hematology and the Raymond G. Perelman Center for Cellular and Molecular Therapeutics at CHOP.

She continues her research into hemophilia, specifically the variants of coagulation factor VIII, to identify novel variants with enhanced function with an eye toward developing gene-based therapeutic approaches for the condition.

Some of Dr. Sabatino's notable accomplishments involve leadership roles and membership in national organizations, including:

  • Serving as co-chair of the NHLBI/NIH State of the Science Workshop, Factor VIII Inhibitors: Generating a National Blueprint for Future Research, Bethesda, MD, 2018
  • Serving as co-chairman of the Scientific and Standardization Committee (SSC) Scientific Subcommittee on Animal, Cellular and Molecular Models, International Society on Thrombosis and Haemostasis, 2013
  • Membership in the Immunology of Gene and Cell Therapy Committee, American Society of Gene and Cell Therapy, 2008-2014


Education and Training

BS, The Ohio State University (Molecular Genetics), 1992

PhD, The George Washington University/National Institutes of Health (Genetics), 2000

Postdoctoral Research Fellow, Children's Hospital of Philadelphia, 2003-2005

Titles and Academic Titles


Research Assistant Professor of Pediatrics

Professional Memberships

American Society of Gene and Cell Therapy, 1998-

American Society of Hematology, 2008-

International Society on Thrombosis and Haemostasis, 2012-

North American Society on Thrombosis and Hemostasis (NASTH), 2013-

Professional Awards

Ruth L. Kirschstein National Research Service Award (F32), 2002

Early Career Investigator Award, Bayer Hemophilia Awards, Bayer Healthcare, LLC, 2009

ASPIRE Hemophilia Research Award, Pfizer, 2012

Publication Highlights

Nguyen GN, George LA, Siner JI, Davidson RJ, Zander CB, Zheng XL, Arruda VR, Camire RM, Sabatino DE. Novel human factor VIII variants with a modified furin cleavage site improve the efficacy of gene therapy for hemophilia A. J Thromb Haemost. 2017 Jan; 15(1):110-121. PMID 27749002
Lange AM, Altynova E, Nguyen G, Sabatino DE. Overexpression of factor VIII after AAV delivery is transiently associated with cellular stress in hemophilia A mice. Mol Ther Methods Clin Dev. 2016 Sep; 3: 16064. PMID 27738645
Sabatino DE, Lange AM, Altynova ES, Sarkar R, Zhou S, Merricks EP, Franck HG, Nichols TC, Arruda VR, Kazazian HH. Efficacy and safety of long-term prophylaxis in severe hemophilia A dogs following liver gene therapy using AAV vectors. Mol.Ther. 2011 Mar; 19(3): 442-449. PMCID: 21081906
Finn JD, Ozelo MC, Sabatino DE, Franck HW, Merricks EP, Crudele JM, Zhou S, Kazazian HH, Lillicrap D, Nichols TC, Arruda VR. Eradication of neutralizing antibodies to factor VIII in canine hemophilia A after liver gene therapy. Blood. 2010 Dec; 116(26):5842-8 PMCID: 20876851
Sabatino DE, Freguia CF, Toso R, Santos A, Merricks EP, Kazazian HH, Nichols TC, Camire RM, Arruda VR. Recombinant canine B-domain-deleted FVIII exhibits high specific activity and is safe in the canine hemophilia A model. Blood. 2009 Nov; 114(20): 4562-4565 PMCID: 19770361