Abstract

The determination of antigen levels and quantitative assays of the biological function of factors VIII and IX in complex mixtures are essential for the success of the projects in this program. The goal of the Coagulation Core is to provide the necessary support and expertise so that these primary objectives are met. In addition to conducting functional assays and immunochemical determinations of antigen levels, the Core will also provide expertise in coagulation and protein chemistry for the further characterization of factor II and IX produced in the various experimental systems used in Project 1-3. These functions of the Core will the availability of appropriate support and expertise for the completion of the aims listed in these projects.

  Funding Agency

Agency
National Institute of Health (NIH)
Institute
National Heart, Lung, and Blood Institute (NHLBI)
Type
Research Program Projects (P01)
Project #
5P01HL064190-03
Application #
6593888
Study Section
Heart, Lung, and Blood Program Project Review Committee (HLBP)
Project Start
2001-12-01
Project End
2002-11-30
Budget Start
Budget End
Support Year
3
Fiscal Year
2002
Total Cost
Indirect Cost

  Institution

Name
Children's Hospital of Philadelphia
Department
Type
DUNS #
073757627
City
Philadelphia
State
PA
Country
United States
Zip Code
19104

  Publications

French, Robert A; Samelson-Jones, Benjamin J; Niemeyer, Glenn P et al. (2018) Complete correction of hemophilia B phenotype by FIX-Padua skeletal muscle gene therapy in an inhibitor-prone dog model. Blood Adv 2:505-508
George, Lindsey A; Sullivan, Spencer K; Giermasz, Adam et al. (2017) Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant. N Engl J Med 377:2215-2227
Gollomp, Kandace; Lambert, Michele P; Poncz, Mortimer (2017) Current status of blood 'pharming': megakaryoctye transfusions as a source of platelets. Curr Opin Hematol 24:565-571
Sim, Xiuli; Poncz, Mortimer; Gadue, Paul et al. (2016) Understanding platelet generation from megakaryocytes: implications for in vitro-derived platelets. Blood 127:1227-33
Marcos-Contreras, Oscar A; Smith, Shannon M; Bellinger, Dwight A et al. (2016) Sustained correction of FVII deficiency in dogs using AAV-mediated expression of zymogen FVII. Blood 127:565-71
Arruda, V R; Samelson-Jones, B J (2016) Gene therapy for immune tolerance induction in hemophilia with inhibitors. J Thromb Haemost 14:1121-34
High, Katherine A; Anguela, Xavier M (2016) Adeno-associated viral vectors for the treatment of hemophilia. Hum Mol Genet 25:R36-41
Siner, Joshua I; Samelson-Jones, Benjamin J; Crudele, Julie M et al. (2016) Circumventing furin enhances factor VIII biological activity and ameliorates bleeding phenotypes in hemophilia models. JCI Insight 1:e89371
Zhang, Nanyan; Zhi, Huiying; Curtis, Brian R et al. (2016) CRISPR/Cas9-mediated conversion of human platelet alloantigen allotypes. Blood 127:675-80
Antony-Debré, Iléana; Manchev, Vladimir T; Balayn, Nathalie et al. (2015) Level of RUNX1 activity is critical for leukemic predisposition but not for thrombocytopenia. Blood 125:930-40

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