Literature DB >> 11449330

Structural and functional characterization of B-domain deleted recombinant factor VIII.

H Sandberg1, A Almstedt, J Brandt, V M Castro, E Gray, L Holmquist, M Lewin, U Oswaldsson, M Mikaelsson, M A Jankowski, M Bond, H A Scoble.   

Abstract

A new high-purity recombinant factor VIII preparation has been developed for the treatment of hemophilia A. Structurally, this factor VIII preparation, B-domain deleted recombinant factor VIII (BDDrFVIII), differs from other recombinant and plasma-derived factor VIII preparations in that most of the B-domain has been deleted. To ensure that BDDrFVIII contains the requisite structural and functional features, it has been subjected to detailed biochemical and biophysical characterization in comparison to the plasma-derived form of factor VIII. Laboratory studies have shown that the primary, secondary, and tertiary structures of BDDrFVIII and the posttranslational modifications are similar to those of the [80 + 90]-kd form of plasma-derived factor VIII. In addition, BDDrFVIII has full biologic activity compared with full-length factor VIII preparations.

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Year:  2001        PMID: 11449330     DOI: 10.1016/s0037-1963(01)90103-9

Source DB:  PubMed          Journal:  Semin Hematol        ISSN: 0037-1963            Impact factor:   3.851


  10 in total

Review 1.  Advances and innovations in haemophilia treatment.

Authors:  Rob Peters; Tim Harris
Journal:  Nat Rev Drug Discov       Date:  2018-06-08       Impact factor: 84.694

2.  BIVV001, a new class of factor VIII replacement for hemophilia A that is independent of von Willebrand factor in primates and mice.

Authors:  Ekta Seth Chhabra; Tongyao Liu; John Kulman; Susannah Patarroyo-White; Buyue Yang; Qi Lu; Douglas Drager; Nancy Moore; Jiayun Liu; Amy M Holthaus; Jurg M Sommer; Ayman Ismail; Deana Rabinovich; Zhan Liu; Arjan van der Flier; Allison Goodman; Chris Furcht; Mark Tie; Tyler Carlage; Randy Mauldin; Terrence M Dobrowsky; Zhiqian Liu; Oblaise Mercury; Lily Zhu; Baisong Mei; Volker Schellenberger; Haiyan Jiang; Glenn F Pierce; Joe Salas; Robert Peters
Journal:  Blood       Date:  2020-04-23       Impact factor: 22.113

3.  Activity of transgene-produced B-domain-deleted factor VIII in human plasma following AAV5 gene therapy.

Authors:  Steffen Rosen; Stefan Tiefenbacher; Mary Robinson; Mei Huang; Jaydeep Srimani; Donnie Mackenzie; Terri Christianson; K John Pasi; Savita Rangarajan; Emily Symington; Adam Giermasz; Glenn F Pierce; Benjamin Kim; Stephen J Zoog; Christian Vettermann
Journal:  Blood       Date:  2020-11-26       Impact factor: 22.113

4.  Minimal modification in the factor VIII B-domain sequence ameliorates the murine hemophilia A phenotype.

Authors:  Joshua I Siner; Nicholas P Iacobelli; Denise E Sabatino; Lacramiora Ivanciu; Shangzhen Zhou; Mortimer Poncz; Rodney M Camire; Valder R Arruda
Journal:  Blood       Date:  2013-01-31       Impact factor: 22.113

5.  Development and characterization of lipidic cochleate containing recombinant factor VIII.

Authors:  Razvan D Miclea; Prashant R Varma; Aaron Peng; Sathy V Balu-Iyer
Journal:  Biochim Biophys Acta       Date:  2007-08-19

Review 6.  Turoctocog alfa (NovoEight®)--from design to clinical proof of concept.

Authors:  Mirella Ezban; Knud Vad; Marianne Kjalke
Journal:  Eur J Haematol       Date:  2014-05-28       Impact factor: 2.997

7.  A single chain variant of factor VIII Fc fusion protein retains normal in vivo efficacy but exhibits altered in vitro activity.

Authors:  Yang Buyue; Tongyao Liu; John D Kulman; Garabet G Toby; George D Kamphaus; Susannah Patarroyo-White; Qi Lu; Thomas J Reidy; Baisong Mei; Haiyan Jiang; Glenn F Pierce; Jurg M Sommer; Robert T Peters
Journal:  PLoS One       Date:  2014-11-21       Impact factor: 3.240

Review 8.  Immune Responses to Plasma-Derived Versus Recombinant FVIII Products.

Authors:  Flora Peyvandi; Syna Miri; Isabella Garagiola
Journal:  Front Immunol       Date:  2021-01-22       Impact factor: 7.561

9.  Molecular determinants of the factor VIII/von Willebrand factor complex revealed by BIVV001 cryo-electron microscopy.

Authors:  James R Fuller; Kevin E Knockenhauer; Nina C Leksa; Robert T Peters; Joseph D Batchelor
Journal:  Blood       Date:  2021-05-27       Impact factor: 22.113

10.  Biochemical and functional characterization of a recombinant monomeric factor VIII-Fc fusion protein.

Authors:  R T Peters; G Toby; Q Lu; T Liu; J D Kulman; S C Low; A J Bitonti; G F Pierce
Journal:  J Thromb Haemost       Date:  2013-01       Impact factor: 5.824

  10 in total

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