NBDF funds a broad range of research programs that seek to increase our understanding of the science behind bleeding disorders, how they affect people's lives, and pathways to better treatments and cures.

Role of Protein Disulfide Isomerase in Prothrombin Activation

Role of Protein Disulfide Isomerase in Prothrombin Activation

Year:
-
Grants:
Judith Graham Pool Postdoctoral Research Fellowship
Prothrombin Activation
Author(s):
Sol Schulman
Per Dr. Schulman, the JGP enabled him to dedicate time to hemophilia research, and more importantly, demonstrated to his department that what he is doing is extremely important to external funding sources. Dr. Schulman has never been formally involved with a Hemophilia Treatment Center, however, his institution has an entire division of hemostasis and thrombosis with clinical and research activity. As a note in proof, I would like to add that the generous support of the NHF has also been instrumental in helping me to establish a viable career path as a physician-scientist focused on the biology and treatment of disorders of hemostasis. Recognition by the NHF with this award has helped me to secure additional protected time and institutional commitment to continue along this career path at Beth Israel Deaconess Medical Center.  I wanted to thank the NHF again, the award has helped me to gain recognition and commitment from my home institution that I expect will eventually enable me to launch an independent career with a focus on bleeding disorders.
A Multi-System Evaluation of von Willebrand Factor Function in Type I von Willebrand Disease Mutations

A Multi-System Evaluation of von Willebrand Factor Function in Type I von Willebrand Disease Mutations

Year:
-
Grants:
Judith Graham Pool Postdoctoral Research Fellowship
Von Willebrand Disease
Author(s):
Christopher Ng

Dr. Christopher Ng was a pediatric hematology/oncology fellow at the University of Colorado - Anschutz Medical Campus. Dr. Ng attended medical school at the Keck School of Medicine at the University of Southern California and completed his pediatrics residency at the University of Washington/Seattle Children's Hospital. Dr. Ng received the NHF-Baxalta Clinical Fellowship in 2013. Dr. Ng's 2015 JGP research fellowship award project focused on a multi-system evaluation of von Willebrand factor function in Type 1 von Willebrand Disease mutations.

Mechanisms and Therapeutic Strategies Targeting TAFI-mediated Vascular Remodeling in Hemophilic Arthropathy

Mechanisms and Therapeutic Strategies Targeting TAFI-mediated Vascular Remodeling in Hemophilic Arthropathy

Year:
-
Grants:
Judith Graham Pool Postdoctoral Research Fellowship
Hemophilic Arthropathy
Pain
Author(s):
Tine L. Wyseure

Dr. Tine Wyseure obtained her Master’s degree in Drug Discovery and Development, and earned her Ph.D. in Pharmaceutical Sciences at the University of Leuven, Belgium. Since 2015, she has been a research associate in the lab of Dr. Laurent Mosnier at The Scripps Research Institute in San Diego. Dr. Wyseure’s 2016 JGP research fellowship award project is focused on investigating the effects of impaired TAFI activation in hemophilia on the progression of hemophilic joint disease. The lack of active TAFI worsens joint bleeding and chronic inflammation and drives the striking development of fragile blood vessels in diseased joints. In search of the missing link, Dr. Wyseure has discovered a novel paradigm on how the formation of new blood vessels is controlled by TAFI and suggests that patients with hemophilia may lack this control switch, causing the formation of unstable and leaky blood vessels.

Understanding the Loss of Perivascular Tissue Factor during Angiogenesis in Hemophilia

Understanding the Loss of Perivascular Tissue Factor during Angiogenesis in Hemophilia

Year:
-
Grants:
Nicholas Cirelli Family
Judith Graham Pool Postdoctoral Research Fellowship
Hemophilia A (Factor VIII/F8)
Hemophilia B (Factor IX/F9)
Author(s):
Laura Sommerville

Dr. Laura Sommerville graduated cum laude from Messiah College and then obtained her MS and PhD degrees in cellular and molecular biology from Temple University. Her graduate work and doctoral dissertation produced several awards and publications in peer reviewed publications. She has been a postdoctoral fellow in the laboratory of Dr. Maureane Hoffman at Duke University since July 2014. Dr. Sommerville's 2015 JGP research fellowship award project is on understanding the loss of perivascular tissue factor during angiogenesis in hemophilia.

A Comprehensive and Unbiased Screen of ADAMTS13 Substrate Specificity

A Comprehensive and Unbiased Screen of ADAMTS13 Substrate Specificity

Year:
-
Grants:
Judith Graham Pool Postdoctoral Research Fellowship
Von Willebrand Disease
Author(s):
Colin Kretz
Per Dr. Kretz, the JGP award came at an ideal point in his career. Through the JGP Program, the support of the NHF plays an important role in developing young and promising research in hematology and bleeding disorders. He is truly grateful for the support of the NHF during his training and considers the award to be an important moment in his career. The work supported by the JGP Fellowship led to two publications, one in PNAS and another in Scientific reports.
Mechanoregulation of von Willebrand Factor Inhibition and Activation

Mechanoregulation of von Willebrand Factor Inhibition and Activation

Year:
-
Grants:
Rueleen Kapsch
Judith Graham Pool Postdoctoral Research Fellowship
Von Willebrand Disease
Author(s):
Hongxia Fu

Per Dr. Fu, the JGP provided her with the opportunity to work on hemophilia, as well as von Willebrand disease. After the completion of her JGP project, Dr. Fu remained in the bleeding disorder field.

Molecular Basis of Procofactor to Cofactor Activation in FVIII

Molecular Basis of Procofactor to Cofactor Activation in FVIII

Year:
-
Grants:
Bob and Margaret Carton
Judith Graham Pool Postdoctoral Research Fellowship
Hemophilia A (Factor VIII/F8)
Author(s):
Sudharsan Parthasarathy

Dr. Parthasarathy's research will tackle two important biological issues in coagulation - namely how procofactor FVIII converts to the active cofactor form (FVIIIa) and binds to IX and X, and the location of FVIII in generating the active Xase complex. Results from this study will provide molecular and biochemical insights into the role of FVIIIa in regulating hemostasis and further elucidate the interactions between coagulation complexes. Dr. Parthasarathy obtained his Masters in Biotechnology from Jawaharlal Nehru University in New Delhi, India and received his Ph.D. in Biochemistry from the University of Kansas in 2011. He has been a postdoctoral researcher in the lab of Dr. Rodney Camire at The Children's Hospital of Philadelphia since July 2011. This award has been made possible through a generous donation from Hemophilia of Georgia, Inc.

In vivo Imaging of Bleeds in Hemophilia B

In vivo Imaging of Bleeds in Hemophilia B

Year:
-
Grants:
Judith Graham Pool Postdoctoral Research Fellowship
Hemophilia B (Factor IX/F9)
Hemophilic Arthropathy
Pain
Author(s):
Elizabeth Chappell

Dr. Chappell's research project will develop a model for characterizing bleeding in hemophilia and particularly in joints. Using mouse models of hematoma formation and knee joint bleeding, Dr. Chappell will use 3D fluorescent imaging technology in "living" hemophilia B mice to better trace bleeding over time- from induction of a bleed to its resolution. This project will provide additional insights on the basic science underlying hemophilic bleeds, not to mention the optimal interventions and timing of treatment to potentially prevent damage caused by bleeds. Dr. Chappell earned her Ph.D. in Pharmaceutical Sciences from UNC Chapel Hill in 2013. She will pursue her research under the mentorship of Dr. Dougald Monroe, Professor in the Division of Hematology/Oncology, UNC School of Medicine and the UNC McAllister Heart Institute.

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Understanding the Interplay between Electron Transfer and VKOR Supported Blood Coagulation

Year:
-
Grants:
Nicholas Cirelli Family
Judith Graham Pool Postdoctoral Research Fellowship
Vitamin K
Author(s):
Wei Cheng
Induced Pluripotent Stem Cell-Derived Platelet Therapy for Hemophilia A

Induced Pluripotent Stem Cell-Derived Platelet Therapy for Hemophilia A

Year:
-
Grants:
Judith Graham Pool Postdoctoral Research Fellowship
Platelets
Hemophilia A (Factor VIII/F8)
Author(s):
Ji Yoon Noh

Dr. Noh's research will utilize induced pluripotent stem cells (iPSC) and manipulate them in vitro to expand production of megakaryocytes and platelets that express therapeutic proteins, including FVIII. The project will further determine whether this system of autologous platelets which overexpress FVIII can be delivered directly to the site of injury and hemorrhage, thereby circumventing and evading neutralization by alloantibody inhibitors in hemophilia A. Dr. Noh received her Ph.D. in Preventive Pharmacology from Seoul National University in South Korea. She has been a postdoctoral fellow in Dr. Mitchell Weiss' lab at The Children's Hospital of Philadelphia since 2012. Dr. Noh is currently being mentored in this JGP project by Dr. Mortimer Poncz at CHOP.

In vivo Selection of Hematopoietic Stem Cells that are Genetically-Modified to Express Platelet-FVIII for Hemophilia A Gene Therapy

In vivo Selection of Hematopoietic Stem Cells that are Genetically-Modified to Express Platelet-FVIII for Hemophilia A Gene Therapy

Year:
-
Grants:
Judith Graham Pool Postdoctoral Research Fellowship
Gene Therapy
Platelets
Hemophilia A (Factor VIII/F8)
Author(s):
Yingyu Chen

The goal of Dr. Chen's research is to examine a method for selectively expanding hematopoietic stem cells expressing the factor VIII transgene. She will also examine the immune consequences of this approach, based on the idea that gene transfer in platelets evades immune recognition. This research has the potential to elicit important clues to developing an approach for gene therapy of hemophilia A and hemophilia A with inhibitors.

Dr. Chen earned a PhD in hematology from Fujian Medical University in China. She already has more than 27 papers published in the Chinese medical literature. Her research in hemophilia and gene therapy will be under the mentorship of Dr. Qizhen Shi, MD, PhD, Associate Investigator at the Blood Research Institute and Assistant Professor of Pediatric Hematology at the Medical College of Wisconsin.

Biochemical Characterization of Vitamin K Epoxide Reductase

Biochemical Characterization of Vitamin K Epoxide Reductase

Year:
-
Grants:
Judith Graham Pool Postdoctoral Research Fellowship
Vitamin K
Author(s):
Brian Ingram
In the long-term, Dr. Ivanciu's goals include the establishment of an independent research laboratory to focus on better understanding the pathological consequences of disruptions in the hemostatic balance. She believes that, as a past JGP Fellow, she is in a unique position to make meaningful contributions to the field of hematology and possibly help advance new therapies for the treatment of hemophilia.
FXa Variants for Treatment of Hemophilia

FXa Variants for Treatment of Hemophilia

Year:
-
Grants:
Judith Graham Pool Postdoctoral Research Fellowship
Hemophilia A (Factor VIII/F8)
Hemophilia B (Factor IX/F9)
Author(s):
Lacramioara Ivanciu
Per Dr. Ivanciu, the JGP award was the starting point of her career in the field of hemophilia. By receiving this award, she was able to advance her understanding of bleeding disorders and gained the necessary experience in state-of-the-art techniques in animals, molecular biology and biochemistry of clotting factors. All of this has prepared her for the next phase of her career.
Fine Structure-Function Mapping VWF-FVIII Interaction

Fine Structure-Function Mapping VWF-FVIII Interaction

Year:
-
Grants:
Judith Graham Pool Postdoctoral Research Fellowship
Von Willebrand Disease
Hemophilia A (Factor VIII/F8)
Author(s):
Andrew Yee

Dr. Yee earned his undergraduate Credentials from Rensselaer Polytechnic Institute before receiving his doctorate from Rice University. His eventual goal is to operate an independent research laboratory in the field of bioengineering with a focus on understanding molecular mechanisms of hemostasis, thrombosis and vascular diseases. Dr. Yee's research project deals with factor VIII and its adhesion to its chaperone protein, von Willebrand factor. In these experiments, fragments of von Willebrand factor are screened for optimal factor VIII stabilization. By further studying the architecture of the "Factor VIII-von Willebrand Factor" complex, Dr. Yee seeks to gain insight to improve designs of novel therapeutics.

Regulatory Mechanisms in the Activation of Blood Coagulation Factors V and VIII

Year:
-
Grants:
Judith Graham Pool Postdoctoral Research Fellowship
Factor V
Hemophilia A (Factor VIII/F8)
Author(s):
Mettine Bos
Junjiang_Sun

Hemophilic Arthropathy: Gene Delivery Vectors for Determining Mechanisms and Therapy

Year:
-
Grants:
Judith Graham Pool Postdoctoral Research Fellowship
Gene Therapy
Hemophilic Arthropathy
Pain
Author(s):
Jun-Jiang Sun

Targeting Dendritic Cells to Induce Immune Tolerance to Factor VIII

Year:
-
Grants:
Judith Graham Pool Postdoctoral Research Fellowship
Hemophilia A (Factor VIII/F8)
Inhibitors
Author(s):
Ruijin Su

Proficient AAV Vectors for the Treatment of Hemophilia B (2007)

Year:
-
Grants:
Judith Graham Pool Postdoctoral Research Fellowship
Hemophilia B (Factor IX/F9)
Gene Therapy
Author(s):
Jyoti Mathur

Identification of Chemical Modifiers of Coagulation

Year:
-
Grants:
Judith Graham Pool Postdoctoral Research Fellowship
Hemophilia A (Factor VIII/F8)
Hemophilia B (Factor IX/F9)
Author(s):
David Buchner

Proficient AAV Vectors for the Treatment of Hemophilia B (2005)

Year:
-
Grants:
Judith Graham Pool Postdoctoral Research Fellowship
Hemophilia B (Factor IX/F9)
Gene Therapy
Author(s):
Michael Herbert