An improved vector for high-level, consistent retroviral transgene expression in human thymocytes after competitive reconstitution from transduced peripheral blood stem cells.


One problem in hematopoietic stem cell (HSC)-based gene therapy is the low-level, and often transient, transgene expression in progeny cells in vivo. Here we have evaluated retroviral vector designs for improved long-term in vivo transgene expression levels in thymocytes recovered after transplantation of gene-modified HSCs. First, several vector designs… (More)


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