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.

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

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.

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
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
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

Purification and Characterization of Vitamin K Epoxide Reductase

Year:
-
Grants:
Judith Graham Pool Postdoctoral Research Fellowship
Vitamin K
Author(s):
Tao Li

Gene Transfer of Factor VIII to the Liver in Vivo

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

Structure of Factor VIII’s C Domains and their Epitopes

Year:
-
Grants:
Judith Graham Pool Postdoctoral Research Fellowship
Hemophilia A (Factor VIII/F8)
Author(s):
Ting-Chang Hsu

Non-primate Lentiviral Vector-Based Gene Therapy for Hemophilia A

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

Function of Coagulation Factor V in Plasma and Platelet Pools

Year:
-
Grants:
Judith Graham Pool Postdoctoral Research Fellowship
Platelets
Author(s):
Hongmin Sun

Development of a Novel Genomic Transfer Approach for the Treatment of Hemophilia Based on Transduction of Adipose Tissue Using Replication-Defective HSV-1 Vectors

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

Exploiting Mouse Models to Elucidate the Cellular and Molecular Mechanisms Involved in the Pathogenesis of Platelet Storage Pool Deficiencies

Year:
-
Grants:
Judith Graham Pool Postdoctoral Research Fellowship
Platelets
Author(s):
Lisa S. Webb

Gene Therapy for Hemophilia B by Direct Intramuscular Injections of Recombinant Adeno Associated Virus Serotype Vectors

Year:
-
Grants:
Career Development Award
Gene Therapy
Hemophilia B (Factor IX/F9)
Author(s):
Hengjun Chao

Identification of Complement-Type Repeats of LRP Responsible for Interaction with A2 Domain of Factor VIII

Year:
-
Grants:
Judith Graham Pool Postdoctoral Research Fellowship
Hemophilia A (Factor VIII/F8)
Author(s):
Andrey Garunovich Sarafanov

Correction of Hemophilia with Lentiviral Vectors

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