NHF 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.

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Preclinical Development of Nuclease-Free Gene Editing for Lifeling Treatment of Bleeding Disorders

Year:
-
Grants:
Judith Graham Pool Postdoctoral Research Fellowship
Tags:
Gene Therapy
Hemophilia A (Factor VIII/F8)
Hemophilia B (Factor IX/F9)
Author(s):
Calvin J. Stephans
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Identification of a Potential Novel Role for Factor IX Using a Zebrafish Model

Year:
-
Grants:
Judith Graham Pool Postdoctoral Research Fellowship
Tags:
Hemophilia B (Factor IX/F9)
Author(s):
Raghunath Azhwar
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The Epitopes Recognized in the Early Immunue Response to Factor VIII

Year:
-
Grants:
Judith Graham Pool Postdoctoral Research Fellowship
Tags:
Hemophilia A (Factor VIII/F8)
Inhibitors
Author(s):
Seema Patel
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Increasing the efficacy of prophylactic infused FIX in hemophilia B patients by manipulating its binding to collagen IV

Year:
-
Grants:
Judith Graham Pool Postdoctoral Research Fellowship
Tags:
Hemophilia B (Factor IX/F9)
Author(s):
Xuejie Chen
Protein Engineering of Plasminogen Activator 1 to Develop Novel Regulators of the Fibrinolytic and Hemostatic Pathways

Protein Engineering of Plasminogen Activator 1 to Develop Novel Regulators of the Fibrinolytic and Hemostatic Pathways

Year:
-
Grants:
Judith Graham Pool Postdoctoral Research Fellowship
Tags:
Plasminogen Activator 1 (PA1)
Author(s):
Laura Haynes

Dr. Laura Haynes received her PhD in biochemistry from the University of Vermont where she studied how flow conditions throughout the vasculature affect thrombin generation, as well as the role of the platelet membrane in modulating the structure/function of the platelet associated prothrombinase complex. Dr. Haynes is currently a research fellow with Dr. David Ginsburg at the University of Michigan. During her JGP fellowship, she will use phage-display technology coupled with high throughput DNA sequencing to make an exhaustive index of the mutations in plasminogen activator inhibitor-1 (PAI-1) that prolong its half-life while not being deleterious in the inhibition of its canonical targets urokinase-type plasminogen activator (uPA) and tissue-type plasminogen activator (tPA). In doing so, she hopes to identify a PAI-1 variant that can downregulate the fibrinolytic process. Dr. Haynes will also implement similar technology to engineer a PAI-1 variant that inhibits activated protein C (APC), thereby prolonging thrombin generation. She hopes that this research will lead to potential therapeutic agents to treat hemophilia and other bleeding disorders.

Identifying novel hemostatic regulation through species-specific studies using zebrafish

Identifying novel hemostatic regulation through species-specific studies using zebrafish

Year:
-
Grants:
Judith Graham Pool Postdoctoral Research Fellowship
Tags:
Hemophilia A (Factor VIII/F8)
Hemophilia B (Factor IX/F9)
Author(s):
Kari Lavik, PhD

Dr. Kari Lavik is a postdoctoral fellow at the University of Michigan in the laboratory of Dr. Jordan Shavit. She received a B.A. in biology from Case Western Reserve University, and her Ph.D. in Biomedical Sciences from The University of Toledo. Her graduate work focused on the study of cancer motility and metastasis through which she became interested in using zebrafish as a model for human disease. In February of 2017, Dr. Lavik joined the Shavit Laboratory in the Department of Pediatrics at the University of Michigan to use zebrafish for the study of bleeding and clotting disorders. For her 2018 JGP fellowship project, she will model hemophilia in the zebrafish, looking for novel species-specific regulators of hemostasis. By delving deeper into the genetic mechanisms that underlie the intrinsic pathway in zebrafish, Dr. Lavik will look for novel gene interactions that can be therapeutically targeted in patients with hemophilia.

Identification, Characterization and Therapeutic Targeting of Key Molecular Markers and Pathways Implicated in the Development of Hemophilic Arthropathy

Identification, Characterization and Therapeutic Targeting of Key Molecular Markers and Pathways Implicated in the Development of Hemophilic Arthropathy

Year:
-
Grants:
Judith Graham Pool Postdoctoral Research Fellowship
Tags:
Hemophilic Arthropathy
Pain
Hemophilia A (Factor VIII/F8)
Hemophilia B (Factor IX/F9)
Author(s):
Esther Cooke

Dr. Esther Cooke received her Ph.D. from the Leeds Institute of Cardiovascular and Metabolic Medicine at the University of Leeds, U.K., where she studied the role of fibrinogen phosphorylation in thrombosis. Dr. Cooke is currently a postdoctoral fellow in the laboratory of Dr. Annette von Drygalski, at the University of California San Diego, and in collaboration with the laboratory of Dr. Laurent Mosnier at the Scripps Research Institute. Dr. Cook's JGP Fellowship project will focus on pathological mechanisms associated with joint bleeding, re-bleeding, and the development of hemophilic arthropathy. Dr. Cooke will perform comprehensive gene expression analyses to explore key molecular markers and pathways that drive soft tissue inflammation and vascular changes in joints after bleeding. In this way, she hopes to identify new therapeutic targets and develop novel treatment strategies to down-regulate these processes, thereby reducing re-bleeding tendency and slowing the progression of hemophilic arthropathy.

Development of Hematopoietic CRISPR/Cas9 Gene Activation for Hemophilia Therapy

Development of Hematopoietic CRISPR/Cas9 Gene Activation for Hemophilia Therapy

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

Dr. Satish Nandakumar is currently a postdoctoral fellow in the laboratory of Dr. Vijay Sankaran at the Boston Children's Hospital. Previously, he did his graduate work at the St. Jude's Children's Research Hospital in Memphis, Tennessee. In his JGP Fellowship project, Dr. Nandakumar aims to develop a novel gene therapy approach for hemophilia that involves activation of the endogenous factor VIII or IX genes within hematopoietic stem cells by taking advantage of the CRISPR/Cas9 gene activation system. This work has the potential to benefit patients with mild hemophilia mutations.

Dissecting the Roles of Non-muscle Myosin IIA in May-Hegglin Platelet Disorders

Dissecting the Roles of Non-muscle Myosin IIA in May-Hegglin Platelet Disorders

Year:
-
Grants:
Judith Graham Pool Postdoctoral Research Fellowship
Tags:
Platelets
Author(s):
Kasturi Pal
Per Dr. Pal, receiving the JGP Fellowship was a major milestone in her academic career and has given her the confidence to apply for future extramural funding.
Structural Biology of Blood Coagulation Proteins and Their Complexes

Structural Biology of Blood Coagulation Proteins and Their Complexes

Year:
-
Grants:
Judith Graham Pool Postdoctoral Research Fellowship
Tags:
Hemophilia A (Factor VIII/F8)
Hemophilia B (Factor IX/F9)
Author(s):
Shekhar Kumar
Per Dr. Kumar, the JGP Fellowship has enabled him to test his ideas related to factor V biology. It has facilitated his scientific training to become increasingly independent in the planning and execution of his research. Important to note, findings generated from these studies have provided new concepts and tools to target factor V for therapeutic purposes. After the completion of his award in 2018, Dr. Kumar would like to continue his career in the field of hematology.
Analysis of Blood Clot Structure and Function in the Presence and Absence of von Willebrand Factor

Analysis of Blood Clot Structure and Function in the Presence and Absence of von Willebrand Factor

Year:
-
Grants:
Judith Graham Pool Postdoctoral Research Fellowship
Tags:
Von Willebrand Disease
Author(s):
Megan S. Rost

Dr. Megan Rost is a postdoctoral fellow at the University of Michigan. She received a B.S in biochemistry and biotechnology from Michigan State University, and her Ph.D. in molecular and developmental biology at the University of Cincinnati - Cincinnati Children's Hospital Medical Center. Her graduate work focused on understanding vascular endothelial development using zebrafish as a model organism. In July 2015, she joined the lab of Dr. Jordan Shavit in the Department of Pediatrics and Hematology/Oncology at University of Michigan. For her 2016 JGP research fellowship project, she will be using the zebrafish model to analyze blood clot structure and function in the presence and absence of von Willebrand Factor. In studying this, Dr. Rost will be elucidating how arterial thrombus formation occurs in the absence of VWF, aiding in uncovering possible new therapeutic targets for VWD treatment.

Role of Protein Disulfide Isomerase in Prothrombin Activation

Role of Protein Disulfide Isomerase in Prothrombin Activation

Year:
-
Grants:
Judith Graham Pool Postdoctoral Research Fellowship
Tags:
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.
Mechanisms of Flow-regulated VWF-platelet Adhesion at Different Length Scales

Mechanisms of Flow-regulated VWF-platelet Adhesion at Different Length Scales

Year:
-
Grants:
Judith Graham Pool Postdoctoral Research Fellowship
Tags:
Von Willebrand Disease
Author(s):
Klaus Bonazza

Klaus Bonazza received his Ph.D. in chemistry from Vienna University of Technology. He is currently a postdoctoral researcher at Boston Children's Hospital and appointed at Harvard Medical School, mentored by Dr. Timothy Springer. His field of interest is the ultra-large concatemeric protein von Willebrand factor (VWF), which accounts for the adaptability of hemostasis to different flow conditions in the blood vessels.

At moderate, physiological flow VWF has a packed, "bird nest's" shape whereas strong elongational flow conditions, occurring downstream of vascular restrictions or injuries, induce a transition to a threat-like, elongated state. On top of this overall unpacking, tensile forces, which are exerted on the chain and transmitted by its A1 domain, cause local conformational changes which activate binding of thrombocyte receptor Glycoprotein Ib (GPIbα) to initiate coagulation. With his JGP fellowship award, Dr. Bonazza will pioneer a new method to obtain structural insights into force dependent VWF unpacking, A1 deformation and GPIbα binding based on hydrogendeuterium exchange under elongational flow conditions.

Understanding the Loss of Perivascular Tissue Factor during Angiogenesis in Hemophilia

Understanding the Loss of Perivascular Tissue Factor during Angiogenesis in Hemophilia

Year:
-
Grants:
Judith Graham Pool Postdoctoral Research Fellowship
Tags:
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 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
Tags:
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
Tags:
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.

Mechanoregulation of von Willebrand Factor Inhibition and Activation

Mechanoregulation of von Willebrand Factor Inhibition and Activation

Year:
-
Grants:
Judith Graham Pool Postdoctoral Research Fellowship
Tags:
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:
Judith Graham Pool Postdoctoral Research Fellowship
Tags:
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.

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
Tags:
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.
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Understanding the Interplay between Electron Transfer and VKOR Supported Blood Coagulation

Year:
-
Grants:
Judith Graham Pool Postdoctoral Research Fellowship
Tags:
Vitamin K
Author(s):
Wei Cheng