Publications by authors named "Mario Abreu Soares Neto"
Article Synopsis
- The study aimed to create recombinant factor IX (FIX) variants that have enhanced clotting activity for hemophilia B treatment.
- Three new FIX variants were developed (FIX-YKALW, FIX-ALL, and FIX-LLW), which had lower antigen expression levels compared to the wild-type but exhibited significantly higher biological activity in clotting assays.
- Among the variants, FIX-YKALW showed the best results in promoting clotting and providing hemostatic protection in mice models, indicating potential for clinical use in protein replacement therapy.
Article Synopsis
- Factor IX (FIX) is a key protein for treating Hemophilia B, and researchers studied its production using two human cell lines: 293T and SK-Hep-1.
- SK-Hep-1 cells produced more biologically active FIX, although they showed slower growth compared to their non-FIX counterparts, while 293T cells did not have their growth affected by gene integration.
- The study indicated that while 293T cells can efficiently carboxylate FIX (important for its activity), only a fraction is active; in contrast, SK-Hep-1 cells secrete high amounts of active protein, making them a promising platform for producing recombinant FIX.
Hemophilia A is caused by a deficiency in coagulation factor VIII. Recombinant factor VIII can be used as an alternative although it is unavailable for most patients. Here, we describe the production of a human recombinant B-domain-deleted FVIII (rBDDFVIII) by the human cell line SK-HEP-1, modified by a lentiviral vector rBDDFVIII was produced by recombinant SK-HEP cells (rSK-HEP) at 1.
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