Efficient and persistent expression of β‐glucuronidase gene in CD34⁺ cells from human umbilical cord blood by retroviral vector

Abstract

We succeeded in efficiently transferring the β‐glucuronidase gene in a retroviral vector to human hematopoietic progenitor cells using a centrifugation enhancement protocol. The transduction efficiency in CFU–GM was highly variable (23–100%) with an average of 66.8%. In the case of BFU–E, efficiency was 83% and 76% in 2 separate experiments. In LTCIC (long‐term culture‐initiating cell), transduction efficiency were 20% and 50% in 2 experiments. The enzymatic activity of β‐glucuronidase in transduced cells were increased above the control level up to 5 wk. Considering that correction of the enzyme deficiency in a small number of hematopoietic cells can be therapeutic for the Sly disease mouse, our data provide encouragement that human trials of gene therapy based on transferring β‐glucuronidase gene to hematopoietic cells may be efficacious.