2011 NHF/Novo Nordisk Career Development Award

Jordan A. Shavit, MD, PhD University of Michigan, Ann Arbor, MI

“Identification of Chemical and Genetic Modifiers of Bleeding Disorders Using a Zebrafish Model System” Dr. Shavit’s research project focuses on better understanding the genetic and molecular pathways that modify or regulate hemostasis through the use of a zebrafish model. Specifically, he will utilize zebrafish genetics to potentially identify the modifier genes that result in reduced or absent bleeding. This innovative proposal may lead to improved therapeutic approaches, especially with regard to screening or potentially tailoring treatments to specific patients. As the recipient of our 2011 NHF/Novo Nordisk Career Development Award, Dr. Shavit will receive support for up to three years. Dr. Shavit received his undergraduate degree in cell and molecular biology from the University of Michigan and both his MD and PhD from Northwestern University. Prior to his appointment as an Assistant Professor of Pediatrics and Communicable Diseases at the University of Michigan in 2009, he spent four years as a postdoctoral research fellow at the University of Michigan under the mentorship of Dr. David Ginsburg. Dr. Shavit is also a former NHF-Baxter Clinical Fellow, having received the award in 2006-2008. The funding for this award is made possible thanks to a generous grant from Novo Nordisk to NHF’s Research Catalyst Fund.

2009-2012 Recipient

Keri C. Smith, Ph.D. University of Texas Health Science Center at Houston

“Identification and Amelioration of T-cell Mediated Inflammatory Cytokines that Contribute to Anti-Factor VIII Inhibitor Formation in Hemophilia A.” Dr. Smith’s research focuses on better understanding the development of factor VIII inhibitors. Specifically, she will focus on the effects of chemical signals, or cytokines, secreted by helper T cells on the development of inhibitor antibodies. She hypothesizes that certain cells, called Th17 cells, play an important role in the development of these antibodies by stimulating inflammation and driving the immune response toward inhibitor production. Dr. Smith received a BS from the University of Delaware before earning her Ph.D. from Montana State University. Prior to her appointment as an Assistant Professor at the University of Texas Medical School at Houston, she spent four years as a postdoctoral research fellow at the University of Michigan.

2008-2009 Recipient

Veronica H. Flood, MD Medical College of Wisconsin

“Studies of VWF Function in VWF-Platelet and VWF-FVIII Interactions” Dr. Flood earned her undergraduate degree from Harvard University in 1995 and her doctorate from Tufts University in 1999. She is board certified in pediatrics as well as pediatric hematology/oncology. She has been serving as an Assistant Professor at the Medical College of Wisconsin, where she has been performing research and seeing patients since 2006. As part of her NHF Career Development Award, Dr. Flood will study the common variants and interactions of the von Willebrand factor molecule as a means to enhance overall understanding and eventually improve upon current methods of von Willebrand disease testing. Using mouse models, she will investigate diagnostic alternatives by examining the interaction between ristocetin and its ability to bind to von Willebrand factor. As Dr. Flood states in her summary, “Improved testing for VWD will prevent patients with normal VWF function from receiving unnecessary treatment, while allowing more accurate assessment of patients with true defects in VWD.”

 

2008 -present             2007-present             2007-present               2007-present               2006-2009               2005–2008             2005–2008                   2004-2007              2004-2007             2004-2007               2004-2007             2003-2006               2003-2006                 2003-2006		Veronica Flood, MD Medical College of Wisconsin Milwaukee, WI   Project Title: Studies of VWF Function in VWF-Platelet and VWF-FVIII Interactions     Alisa Wolberg, Ph.D. University of North Carolina at Chapel Hill Chapel Hill, NC   Project Title: Recombinant Factor VIIa and RL Platelets as a Hemophilia Therapy     Qizhen Shi, MD, Ph.D. Medical College of Wisconsin Milwaukee, WI   Project Title: Immune Response in Platelet-Derived FVIII Gene Therapy of Murine Hemophilia A     Jay Lozier, MD, Ph.D. US Food and Drug Administration Rockville, MD   Project Title: Pharmacogenomics of Hemophilia Therapy: Genetics of Inhibitor Antibody Response     Yashuhiro Ikeda, Ph.D., DVM Mayo Clinic Rochester, MN   Project Title: Self-Regulating HIV Vectors for Hemophilia A Gene Therapy     Donald L. Yee, MD Texas Children’s Cancer Center Houston, Texas   Project Title: Platelets as Modifiers of Phenotype in Hemophilia A     Bradley E. Aouizerat, Ph.D. University of California San Francisco San Francisco, California   Project Title: A Disease-Specific DNA Chip for von Willebrand Disease           Kathleen Brummel-Ziedins, Ph.D. University of Vermont Burlington, Vermont   Project Title: Individualized Phenotypic Prophylaxis for Hemophilia     Suzie Hwang Pun, Ph.D. University of Washington Seattle, Washington   Project Title: Development of Nanoparticles for Non-viral Hemophilia Gene Therapy     Bin Zhang, Ph.D. University of Michigan Ann Arbor, Michigan   Project Title: Regulation of Factor V and VIII Secretion by an ER to Golgi Transport Receptor     Huang-Ge Zhang, DVM, MD, Ph.D. University of Alabama at Birmingham Birmingham, Alabama   Project Title: Depletion of Immune Response to AAV-F.IX     Lingfei Xu, MD Washington University St. Louis, Missouri   Project Title: Retroviral Vector-Mediated Neonatal Gene Therapy for Hemophilia A     Paul E. Monahan, MD University of North Carolina Chapel Hill, North Carolina   Project Title: Double-stranded Adeno-associated Virus (dsAAV) Vectors to Improve Efficacy and                      to Evaluate Safety of Factor IX Gene Therapy     Hiroyuki Nakai, MD, Ph.D. Stanford University School of Medicine Stanford, California   Project Title: Establishing the Factors Responsible for Hepatocyte Permissiveness to AAV Vectors
2002-2004             2002-2005		Yubin Kang, MD University of Iowa College of Medicine Iowa City, Iowa   Project Title: Non-primate Lentiviral Vector-Based Gene Therapy for Hemophilia A     Carol H. Miao, Ph.D. University of Washington Seattle, WA   Project Title: Nonviral Gene Delivery for Hemophilia
2002-2005		Frank Park, Ph.D. Louisiana State University New Orleans, LA   Project Title: Gene Transfer of Factor VIII to the Liver in Vivo   .
2001-2004		Hengjun Chao, MD University of North Carolina Chapel Hill, North Carolina   Project Title: Gene Therapy for Hemophilia B by Direct Intramuscular Injections of Recombinant                      Adeno Associated Virus Serotype Vectors
2000-2003		Steven Wesley Pipe, MD University of Michigan Ann Arbor, Michigan   Project Title: Development and Characterization of Novel Genetically Engineered Factor VIII                      Molecules with Increased Activity and Stability
2000-2003		Roland W. Herzog, Ph.D. The Children’s Hospital of Philadelphia Philadelphia, Pennsylvania   Project Title:  Immunology of Liver-Derived Expression of Factor IX from AAV Vectors
2000-2003		Tal Kafri, M.D., Ph.D. University of North Carolina Chapel Hill, North Carolina   Project Title: Correction of Hemophilia with Lentiviral Vectors