Ontology highlight
ABSTRACT:
SUBMITTER: Koldej RM
PROVIDER: S-EPMC4003467 | biostudies-literature | 2013 Jun
REPOSITORIES: biostudies-literature
Koldej Rachel M RM Carney Gael G Wielgosz Matthew M MM Zhou Sheng S Zhan Jun J Sorrentino Brian P BP Nienhuis Arthur W AW
Human gene therapy. Clinical development 20130620 2
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 a ...[more]