A novel B-domain O-glycoPEGylated FVIII (N8-GP) demonstrates full efficacy and prolonged effect in hemophilic mice models

作者: Henning R. Stennicke , Marianne Kjalke , Ditte M. Karpf , Kristoffer W. Balling , Peter B. Johansen

DOI: 10.1182/BLOOD-2012-01-407494

关键词:

摘要: … In conclusion, N8-GP has the potential to offer efficacious prevention and treatment of … Capping of nonsialydated glycans is performed by addition of excess CMP-SA and ST3GalIII. The …

参考文章(35)
CA Siediecki, BJ Lestini, KK Kottke-Marchant, SJ Eppell, DL Wilson, RE Marchant, Shear-dependent changes in the three-dimensional structure of human von Willebrand factor Blood. ,vol. 88, pp. 2939- 2950 ,(1996) , 10.1182/BLOOD.V88.8.2939.BLOODJOURNAL8882939
L Bi, R Sarkar, T Naas, AM Lawler, J Pain, SL Shumaker, V Bedian, HH Jr Kazazian, Further characterization of factor VIII-deficient mice created by gene targeting: RNA and protein studies. Blood. ,vol. 88, pp. 3446- 3450 ,(1996) , 10.1182/BLOOD.V88.9.3446.BLOODJOURNAL8893446
Di Capua M, Coppola A, De Simone C, Primary prophylaxis in children with haemophilia Vox Sanguinis. ,vol. 6, ,(2008) , 10.2450/2008.0030-08
T. ELM, D. M. KARPF, K. ØVLISEN, H. PELZER, M. EZBAN, M. KJALKE, M. TRANHOLM, Pharmacokinetics and pharmacodynamics of a new recombinant FVIII (N8) in haemophilia A mice. Haemophilia. ,vol. 18, pp. 139- 145 ,(2012) , 10.1111/J.1365-2516.2011.02608.X
Michael Felo, Shawn DeFrees, Nucleotide sugar purification using membranes ,(2006)
Yoshihiro UCHIDA, Yoji TSUKADA, Tsunetake SUGIMORI, Enzymatic Properties of Neuraminidases from Arthrobacter ureafaciens Journal of Biochemistry. ,vol. 86, pp. 1573- 1585 ,(1979) , 10.1093/OXFORDJOURNALS.JBCHEM.A132675
L. THIM, B. VANDAHL, J. KARLSSON, N. K. KLAUSEN, J. PEDERSEN, T. N. KROGH, M. KJALKE, J. M. PETERSEN, L. B. JOHNSEN, G. BOLT, P. L. NØRBY, T. D. STEENSTRUP, Purification and characterization of a new recombinant factor VIII (N8). Haemophilia. ,vol. 16, pp. 349- 359 ,(2010) , 10.1111/J.1365-2516.2009.02135.X
U. MARTINOWITZ, J. BJERRE, B. BRAND, R. KLAMROTH, M. MISGAV, M. MORFINI, E. SANTAGOSTINO, A. TIEDE, D. VIUFF, Bioequivalence between two serum-free recombinant factor VIII preparations (N8 and ADVATE®)--an open-label, sequential dosing pharmacokinetic study in patients with severe haemophilia A. Haemophilia. ,vol. 17, pp. 854- 859 ,(2011) , 10.1111/J.1365-2516.2011.02495.X
W. Antuch, P. Güntert, M. Billeter, T. Hawthorne, H. Grossenbacher, K. Wüthrich, NMR solution structure of the recombinant tick anticoagulant protein (rTAP), a factor Xa inhibitor from the tick Ornithodoros moubata. FEBS Letters. ,vol. 352, pp. 251- 257 ,(1994) , 10.1016/0014-5793(94)00941-4
Jan Egebjerg, Søren Christensen, Cloning, expression and characterization of a sialidase gene from Arthrobacter ureafaciens Biotechnology and Applied Biochemistry. ,vol. 41, pp. 225- 231 ,(2005) , 10.1042/BA20040144