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Structural and functional characterization of B-domain deleted recombinant factor VIII
H Sandberg1, A Almstedt, J Brandt
1Pharmacia AB, Stockholm, Sweden.
Seminars in Hematology
|July 13, 2001
Summary
A novel B-domain deleted recombinant factor VIII (BDDrFVIII) shows structural and functional similarity to plasma-derived factor VIII. This high-purity preparation offers full biologic activity for hemophilia A treatment.
Area of Science:
- Biochemistry
- Hematology
- Protein Engineering
Background:
- Hemophilia A is a bleeding disorder caused by factor VIII deficiency.
- Current treatments include plasma-derived and recombinant factor VIII (FVIII) preparations.
- A B-domain deleted recombinant factor VIII (BDDrFVIII) has been developed.
Purpose of the Study:
- To characterize the biochemical and biophysical properties of BDDrFVIII.
- To compare BDDrFVIII with plasma-derived factor VIII (PDFVIII).
- To confirm the structural integrity and biological activity of BDDrFVIII.
Main Methods:
- Detailed biochemical and biophysical characterization of BDDrFVIII.
- Comparative analysis of primary, secondary, and tertiary structures.
- Assessment of posttranslational modifications and biological activity.
Main Results:
- BDDrFVIII exhibits similar primary, secondary, and tertiary structures to the [80 + 90]-kd form of PDFVIII.
- Posttranslational modifications in BDDrFVIII are comparable to PDFVIII.
- BDDrFVIII demonstrates full biological activity, equivalent to full-length FVIII.
Conclusions:
- BDDrFVIII is a high-purity recombinant factor VIII preparation.
- The structural and functional characteristics of BDDrFVIII are well-preserved.
- BDDrFVIII represents a promising therapeutic option for hemophilia A.