Computationally designed liver-specific transcriptional modules and hyperactive factor IX improve hepatic gene therapy.

Autor: Nair N; Department of Gene Therapy and Regenerative Medicine, Free University of Brussels, Brussels, Belgium;, Rincon MY; Department of Gene Therapy and Regenerative Medicine, Free University of Brussels, Brussels, Belgium; Center for Molecular and Vascular Biology, Department of Cardiovascular Medicine, University of Leuven, Leuven, Belgium;, Evens H; Department of Gene Therapy and Regenerative Medicine, Free University of Brussels, Brussels, Belgium;, Sarcar S; Department of Gene Therapy and Regenerative Medicine, Free University of Brussels, Brussels, Belgium;, Dastidar S; Department of Gene Therapy and Regenerative Medicine, Free University of Brussels, Brussels, Belgium;, Samara-Kuko E; Department of Gene Therapy and Regenerative Medicine, Free University of Brussels, Brussels, Belgium;, Ghandeharian O; Department of Gene Therapy and Regenerative Medicine, Free University of Brussels, Brussels, Belgium;, Man Viecelli H; Department of Pediatrics, Division of Metabolism, University of Zurich, Zurich, Switzerland; and., Thöny B; Department of Pediatrics, Division of Metabolism, University of Zurich, Zurich, Switzerland; and., De Bleser P; Inflammation Research Center, Vlaams Instituut voor Biotechnologie, and Department of Biomedical Molecular Biology, Ghent University, Ghent, Belgium., VandenDriessche T; Department of Gene Therapy and Regenerative Medicine, Free University of Brussels, Brussels, Belgium; Center for Molecular and Vascular Biology, Department of Cardiovascular Medicine, University of Leuven, Leuven, Belgium;, Chuah MK; Department of Gene Therapy and Regenerative Medicine, Free University of Brussels, Brussels, Belgium; Center for Molecular and Vascular Biology, Department of Cardiovascular Medicine, University of Leuven, Leuven, Belgium;

Publikováno v: Blood [Blood] 2014 May 15; Vol. 123 (20), pp. 3195-9. Date of Electronic Publication: 2014 Mar 17.