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.

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
Development of an Institution-Wide, Customizable, Computer-Based, Training Module

Development of an Institution-Wide, Customizable, Computer-Based, Training Module

Year:
-
Grants:
Nursing Excellence Fellowship
Author(s):
Robin Chapman
The purpose of this project is to request support for the development of a computer-based training (CBT) module designed to teach the care and treatment of patients with bleeding disorders to nursing staff in a large, multi-campus pediatric healthcare institution. The module will be designed to exist within an intranet environment accessed by all members of the healthcare institution. Core modules will include a description of each common bleeding disorder, a basic review of processes surrounding the multidisciplinary care of bleeding disorder patients, the involvement of other hospital departments in managing a patient with a bleeding treatment plan (e.g., blood bank, coagulation lab, hemophilia treatment center staff) and medications/factor products routinely used to treat them. The result, a custom-made bleeding disorder CBT, would be available at any time to any staff member on any of the institution's campuses being assigned to care for bleeding disorder patients, thus facilitating nursing education and patient care.
The Prevalence and Risk Factors for Depression in Adults with Hemophilia

The Prevalence and Risk Factors for Depression in Adults with Hemophilia

Year:
-
Grants:
Social Work Excellence Fellowship
Author(s):
Laurel Pennick, Maria Iannone
Despite the fact that adults with hemophilia fit the profile of a population at high risk for experiencing depression, not to mention the significant impact of depression on their overall well-being, there are almost no studies in the literature to document the incidence of depression for these patients. This study is designed to document the prevalence of depression in adults with hemophilia at our treatment center and to evaluate risk factors for depression in these patients as well as patients from Region VIII who have completed the Centers for Disease Control and Prevention's Quality of Life questionnaire. Evaluation of the information collected is expected to show that a significant percentage of adults with hemophilia are experiencing symptoms of depression. The Arizona Hemophilia and Thrombosis Center plans to use this information to apply for future funding to support the development of a depression care management plan, which will be incorporated into standard care.

Platelets as Modifiers of Phenotype in Hemophilia A

Year:
-
Grants:
Career Development Award
Hemophilia A (Factor VIII/F8)
Platelets
Author(s):
Donald L. Yee

A Disease-Specific DNA Chip for von Willebrand Disease

Year:
-
Grants:
Career Development Award
Von Willebrand Disease
Author(s):
Bradley E. Aouizerat

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

Regulation of Factor V and VIII Secretion by an ER to Golgi Transport Receptor

Year:
-
Grants:
Career Development Award
Factor V
Hemophilia A (Factor VIII/F8)
Author(s):
Bin Zhang

Transposon Mediated Gene Therapy of Hemophilia

Year:
-
Grants:
Judith Graham Pool Postdoctoral Research Fellowship
Gene Therapy
Hemophilia A (Factor VIII/F8)
Hemophilia B (Factor IX/F9)
Author(s):
Li Liu

Individualized Phenotypic Prophylaxis for Hemophilia

Year:
-
Grants:
Career Development Award
Hemophilia A (Factor VIII/F8)
Hemophilia B (Factor IX/F9)
Author(s):
Kathleen Brummel-Ziedins

Depletion of Immune Response to AAV-F.IX

Year:
-
Grants:
Career Development Award
Hemophilia B (Factor IX/F9)
Gene Therapy
Author(s):
Huang-Ge Zhang

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

Development of Nanoparticles for Non-viral Hemophilia Gene Therapy

Year:
-
Grants:
Career Development Award
Gene Therapy
Hemophilia A (Factor VIII/F8)
Hemophilia B (Factor IX/F9)
Author(s):
Suzie Hwang Pun

Illinois Amish Community Outreach Project

Year:
-
Grants:
Social Work Excellence Fellowship
Author(s):
Jenny Purdy
Our goal is to establish an outreach clinic in an area that will serve the Amish bleeding disorder community. After the completion of our formal project, we plan to continue to offer our services to the community to provide ongoing education, screening, and testing for individuals with bleeding symptoms or with a family history of a bleeding disorder. Furthermore, we plan to extend our clinical support to the other Amish communities located in downstate Illinois.
Paul Monahan

Double-stranded Adeno-associated Virus (dsAAV) Vectors to Improve Efficacy and to Evaluate Safety of Factor IX Gene Therapy

Year:
-
Grants:
Career Development Award
Gene Therapy
Hemophilia B (Factor IX/F9)
Author(s):
Paul E. Monahan

Paul Monahan is Translational and Clinical Development Lead for Hematology Gene Therapy at Spark Therapeutics. Prior to 2015, Dr. Monahan was Professor of Pediatrics, Hematology/Oncology at the University of North Carolina at Chapel Hill, where he spent more than 20 years as an Investigator in the UNC Gene Therapy Center and treating children with bleeding disorders as an Attending Physician in the Harold Roberts Hemophilia Diagnostic and Treatment Center. He served on several clinical medical and scientific foundations and committees including the Board of Directors of the Hemostasis and Thrombosis Research Society. For ten years he served on the Medical and Scientific Advisory Committee of the National Hemophilia Foundation and as the Region IV Director for the US Hemophilia Treatment Center Network (CDC and MCHB). His basic science laboratory maintained a research focus on gene therapy for hemophilia as well as animal models for the study of hemophilic bone and joint disease, inhibitors in hemophilia B, and novel therapies from 1996 through 2016. In 2013 the National Hemophilia Foundation awarded Monahan the NHF Leadership in Research Award. Prior to joining Spark in Spring 2018, he performed preclinical research development and coordinated clinical trial initiation of hemophilia B and hemophilia A gene therapy trials in a collaborative partnership with Asklepios Biopharmaceuticals and as Medical Lead at Baxalta/Shire.

Retroviral Vector-Mediated Neonatal Gene Therapy for Hemophilia A

Year:
-
Grants:
Career Development Award
Gene Therapy
Hemophilia A (Factor VIII/F8)
Author(s):
Lingfei Xu

Development of Self-Complimentary (SC) Adeno-Associated Virus Type 5 (AAV-5) Vectors for Gene Therapy of Hemophilia B

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

Mechanism for the Ectopic Expression of Galgt2 in Endothelial Cells

Year:
-
Grants:
Judith Graham Pool Postdoctoral Research Fellowship
Von Willebrand Disease
Author(s):
Jill Marie Johnson

Establishing the Factors Responsible for Hepatocyte Permissiveness to AAV Vectors

Year:
-
Grants:
Career Development Award
Gene Therapy
Hemophilia A (Factor VIII/F8)
Hemophilia B (Factor IX/F9)
Author(s):
Hiroyuki Nakai
Christine_Kempton

Platelet Mechanism Regulating Thrombin Generation

Year:
-
Grants:
Judith Graham Pool Postdoctoral Research Fellowship
Platelets
Author(s):
Christine L. Kempton

Per Dr. Kempton, the JGP assisted her in determining the direction of her future research. As a result of her JGP project, Dr. Kempton was published in ATVB and published several abstracts.

Nonviral Gene Delivery for Hemophilia

Year:
-
Grants:
Career Development Award
Gene Therapy
Hemophilia A (Factor VIII/F8)
Hemophilia B (Factor IX/F9)
Author(s):
Carol H. Miao