Journal article

Comparison of Insulators and Promoters for Expression of the Wiskott-Aldrich Syndrome Protein Using Lentiviral Vectors

Rachel M Koldej, Gael Carney, Matthew M Wielgosz, Sheng Zhou, Jun Zhan, Brian P Sorrentino, Arthur W Nienhuis

HUMAN GENE THERAPY CLINICAL DEVELOPMENT | MARY ANN LIEBERT, INC | Published : 2013

Abstract

Gene therapy for the treatment of Wiskott-Aldrich syndrome (WAS) presents an alternative to the current use of allogeneic bone marrow transplantation. We describe the development of a self-inactivating lentiviral vector containing chromatin insulators for treatment of WAS and compare a gammaretroviral (MND), human cellular (EF1α), and the human WASp gene promoter for expression patterns in vivo during murine hematopoiesis using the green fluorescent protein (GFP) marker. Compared with the EF1α and the WASp promoters, expression from the MND promoter in mouse transplant recipients was much higher in all lineages examined. Importantly, there was sustained expression in the platelets of seconda..

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