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Tissue-specific transcription factors contribute to diverse cellular functions in mammals. For example, the gene for beta globin, a major component of hemoglobin, is present in all cells of the body. However, it is only expressed in red blood cells because the transcription factors that can bind to the promoter sequences of the beta globin gene are only expressed in these cells. Tissue-specific transcription factors also ensure that mutations in these factors may impair only the function of...
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Helical Organization of Blood Coagulation Factor VIII on Lipid Nanotubes
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Extended Half-Life Factor VIII and Factor IX Preparations.

Lukas Graf1

  • 1Center for Laboratory Medicine, Hemophilia and Hemostasis Center, St. Gallen, Switzerland.

Transfusion Medicine and Hemotherapy : Offizielles Organ Der Deutschen Gesellschaft Fur Transfusionsmedizin Und Immunhamatologie
|May 17, 2018
PubMed
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New extended half-life factor VIII and factor IX therapies improve hemophilia treatment. However, current lab assays may not accurately monitor these advanced factor concentrates.

Keywords:
Extended half-lifeFactor concentratesHemophilia A and BProphylaxis

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Area of Science:

  • Hematology
  • Biotechnology
  • Pharmacology

Background:

  • Several extended half-life (EHL) factor VIII and factor IX products have been developed and approved.
  • These EHL products utilize techniques such as Fc fusion, albumin fusion, PEGylation, and protein sequence modification to prolong their therapeutic effect.

Purpose of the Study:

  • To review the development and implications of extended half-life factor VIII and factor IX concentrates.
  • To highlight the challenges these novel therapies present for coagulation laboratory monitoring.

Main Methods:

  • Review of recent scientific literature on extended half-life factor products.
  • Analysis of pharmacokinetic data and clinical outcomes.
  • Evaluation of current coagulation laboratory assays in the context of EHL products.

Main Results:

  • EHL factor IX products show 4- to 6-fold half-life extension.
  • EHL factor VIII products demonstrate 1.5- to 2-fold half-life extension, limited by von Willebrand factor interaction.
  • Both EHL factor VIII and IX products enhance prophylactic therapy for hemophilia A and B.

Conclusions:

  • Extended half-life factor concentrates have significantly improved hemophilia prophylaxis.
  • Current coagulation laboratory assays may not be suitable for accurate monitoring of EHL factor therapies, necessitating further research and development.