In This Section
Dr. Samelson-Jones is a pediatric hematologist dedicated to improving the lives of children with bleeding and clotting disorders. His research focuses on gene therapy for hemophilia, the biochemical basis of coagulation, and the immune responses to hemophilia therapies.
Dr. Samelson-Jones is a pediatric hematologist dedicated to improving the lives of children with bleeding and clotting disorders. He received his doctorate training in protein biophysics at Albert Einstein College of Medicine where he provided the first molecular description of the interaction between nitric oxide and indoleamine 2,3-dioxygenase, a critical enzyme in immune tolerance. He completed clinical training at the Children’s National Medical Center and Children’s Hospital of Philadelphia.
His research at CHOP helped establish the gain-of-function coagulation factor IX variant Padua as the standard transgene for gene therapy for hemophilia B. He was the first to characterize the biochemical basis of the hyperactivity of factor IX Padua, and he helped run the first successful clinical gene therapy trial using factor IX Padua. The first regulatory approved gene therapeutics for hemophilia B are anticipated to use factor IX Padua.
Dr. Samelson-Jones has also studied other approaches to improve gene therapy for hemophilia. He delineated a deleterious interaction between the intracellular protease furin and coagulation factor VIII and demonstrated that bioengineering factor VIII to avoid furin improves gene therapy for hemophilia A. He has also investigated the consequences of immunosuppression on adeno associated viral vector gene therapy and established that concomitant intensive T cell directed immunosuppression and vector administration increases the risk of immune complications.
Education and Training
BA, Amherst College (Physics), 2001
MD, Albert Einstein College of Medicine, 2009
PhD, Albert Einstein College of Medicine (Biophysics), 2009
Pediatric Residency, Children's National Medical Center, 2012
Hematology and Oncology Fellowship, Children's Hospital of Philadelphia, 2015
Titles and Academic Titles
Assistant Professor of Pediatrics
Phi Beta Kappa, 2001-
Sigma Xi, 2001-
American Academy of Pediatrics, 2009-
American Society of Hematology, 2012-
American Society of Pediatric Hematology and Oncology, 2012-
The American Board of Pediatrics, 2013-
Hemostasis and Thrombosis Research Society, 2014-
International Society of Thrombosis and Haemostasis, 2015-
American Society of Gene and Cell Therapy, 2015-
The American Board of Pediatrics and its Sub-board of Pediatric Hematology-Oncology, 2017-
The Basset Physics Prize, 1999
Dean of Faculty Fellowship for Student Research, 2000
The William Warren Stifler Physics Prize, 2001
Society of Porphyrins and Phthalocyanines Student Assistance Grant, 2006
Medical Student Teaching Award, 2011
Thrombosis-Hemostasis Summit of North America (THSNA) Travel Award, 2014
Children’s Hospital of Philadelphia Research Poster Day Prize, 2015
Outstanding Poster Presentation Award, American Society of Gene and Cell Therapy, 2018
Thrombosis-Hemostasis Summit of North America (THSNA) Young Investigator Award, 2020
Rational Development of Bioengineered Factor IX Variants
Work in this proposal would develop and characterize the biochemistry of hyperactive FIX variants, extensively evaluate their efficacy as protein and gene therapeutics for hemophilia B in a large gene deletion hemophilia B mouse model, and evaluate their immunogenicity in several distinct hemophilia B mouse models to test the role of the underlying FIX-gene mutation on tolerance to FIX variants.
Characterization of the Functional Repertoire and ontogeny of FVIII Humor Response Across Species
The goal of this project is to characterize, in depth, the Factor VIII inhibitor response in humans and dogs with severe Hemophilia A and define the role of emerging B cell survival cytokine.
New Technologies for Defining the Factor VIII Inhibitor Response
University of Pennsylvania Hemophilia Treatment Center
The goal of this proposal is to establish novel technologies that will enable comprehensive study of FVIII-specific B cells and antibodies.
PI: Samelson-Jones and Bhoj