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Updated: Jun 28, 2026
Constitutive and Inducible Systems for Genetic In Vivo Modification of Mouse Hepatocytes Using Hydrodynamic Tail Vein Injection
Published on: February 2, 2018
[Non-viral gene transfer results in therapeutic factor IX levels in haemophilia B mice]
J Schüttrumpf1, P Milanov, S Roth
1DRK-Blutspendedienst Baden Württemberg - Hessen gGmbH, Institut für Transfusionsmedizin und Immunhämatologie, Klinikum der Johann Wolfgang Goethe Universität, Sandhofstr. 1, 60528 Frankfurt am Main, Germany. j.schuettrumpf@blutspende.de
Unlabelled:
Safety issues concerning the risk of malignancy formation and immune response to viral vectors were raised in initial gene therapy trials. In contrast, non-viral gene delivery methods have long been offside. We therefore explore a non-viral gene transfer approach for the treatment of hemophilia B.
Methods:
First, we constructed a strong liver-specific expression plasmid for human factor IX (FIX). Next, we tested the vector by injecting two doses under hydrodynamic conditions into the tail veins of FIX knockout mice.
Results:
A single injection resulted in an increase in FIX expression over 100% of normal plasma levels. The FIX resulted fully functional. Further, no anti-FIX antibodies were observed and expression levels were vector dose dependent.
Conclusion:
The high expression obtained in small animals give hope for further development of non-viral gene transfer for the treatment of hemophilia B in humans.
