The future of recombinant coagulation factors

E. L. Saenko; N. M. Ananyeva; M. Shima; C. A.E. Hauserj; S. W. Pipe

doi:10.1046/j.1538-7836.2003.00196.x

The future of recombinant coagulation factors

E. L. Saenko^*, N. M. Ananyeva, M. Shima, C. A.E. Hauserj, S. W. Pipe

^*Corresponding author for this work

Research output: Contribution to journal › Review article › peer-review

40 Scopus citations

Abstract

Hemophilias A and B are X chromosome-linked bleeding disorders, which are mainly treated by repeated infusions of factor (F)VIII or FIX, respectively. In the present review, we specify the limitations in expression of recombinant (r)FVIII and summarize the bioengineering strategies that are currently being explored for constructing novel rFVIII molecules characterized by high efficiency expression and improved functional properties. We present the strategy to prolong FVIII lifetime by disrupting FVIII interaction with its clearance receptors and demonstrate how construction of human-porcine FVIII hybrid molecules can reduce their reactivity towards inhibitory antibodies. While the progress in improving rFIX is impeded by low recovery rates, the authors are optimistic that the efforts of basic science may ultimately lead to higher efficiency of replacement therapy of both hemophilias A and B.

Original language	English (US)
Pages (from-to)	922-930
Number of pages	9
Journal	Journal of Thrombosis and Haemostasis
Volume	1
Issue number	5
DOIs	https://doi.org/10.1046/j.1538-7836.2003.00196.x
State	Published - May 2003
Externally published	Yes

Keywords

Bioengineering
Hemophilia
Recombinant factor IX
Recombinant factor VIII
Replacement therapy

ASJC Scopus subject areas

Hematology

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10.1046/j.1538-7836.2003.00196.x

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title = "The future of recombinant coagulation factors",

abstract = "Hemophilias A and B are X chromosome-linked bleeding disorders, which are mainly treated by repeated infusions of factor (F)VIII or FIX, respectively. In the present review, we specify the limitations in expression of recombinant (r)FVIII and summarize the bioengineering strategies that are currently being explored for constructing novel rFVIII molecules characterized by high efficiency expression and improved functional properties. We present the strategy to prolong FVIII lifetime by disrupting FVIII interaction with its clearance receptors and demonstrate how construction of human-porcine FVIII hybrid molecules can reduce their reactivity towards inhibitory antibodies. While the progress in improving rFIX is impeded by low recovery rates, the authors are optimistic that the efforts of basic science may ultimately lead to higher efficiency of replacement therapy of both hemophilias A and B.",

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author = "Saenko, {E. L.} and Ananyeva, {N. M.} and M. Shima and Hauserj, {C. A.E.} and Pipe, {S. W.}",

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AU - Ananyeva, N. M.

AU - Shima, M.

AU - Hauserj, C. A.E.

AU - Pipe, S. W.

PY - 2003/5

Y1 - 2003/5

N2 - Hemophilias A and B are X chromosome-linked bleeding disorders, which are mainly treated by repeated infusions of factor (F)VIII or FIX, respectively. In the present review, we specify the limitations in expression of recombinant (r)FVIII and summarize the bioengineering strategies that are currently being explored for constructing novel rFVIII molecules characterized by high efficiency expression and improved functional properties. We present the strategy to prolong FVIII lifetime by disrupting FVIII interaction with its clearance receptors and demonstrate how construction of human-porcine FVIII hybrid molecules can reduce their reactivity towards inhibitory antibodies. While the progress in improving rFIX is impeded by low recovery rates, the authors are optimistic that the efforts of basic science may ultimately lead to higher efficiency of replacement therapy of both hemophilias A and B.

AB - Hemophilias A and B are X chromosome-linked bleeding disorders, which are mainly treated by repeated infusions of factor (F)VIII or FIX, respectively. In the present review, we specify the limitations in expression of recombinant (r)FVIII and summarize the bioengineering strategies that are currently being explored for constructing novel rFVIII molecules characterized by high efficiency expression and improved functional properties. We present the strategy to prolong FVIII lifetime by disrupting FVIII interaction with its clearance receptors and demonstrate how construction of human-porcine FVIII hybrid molecules can reduce their reactivity towards inhibitory antibodies. While the progress in improving rFIX is impeded by low recovery rates, the authors are optimistic that the efforts of basic science may ultimately lead to higher efficiency of replacement therapy of both hemophilias A and B.

KW - Bioengineering

KW - Hemophilia

KW - Recombinant factor IX

KW - Recombinant factor VIII

KW - Replacement therapy

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The future of recombinant coagulation factors

Abstract

Keywords

ASJC Scopus subject areas

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