EFFICIENT AND PERSISTENT EXPRESSION OF BETA-GLUCURONIDASE GENE IN CD34(-CORD BLOOD BY RETROVIRAL VECTOR() CELLS FROM HUMAN UMBILICAL)

We succeeded in efficiently transferring the beta-glucuronidase gene i n a retroviral vector to human hematopoietic progenitor cells using a centrifugation enhancement protocol. The transduction efficiency in CF U-GM was highly variable (23-100%) with an average of 66.8%. In the ca se of BFU-E, efficiency was 83 % and 76 % in 2 separate experiments. I n LTCIC (long-term culture-initiating cell), transduction efficiency w ere 20 % and 50 % in 2 experiments. The enzymatic activity of beta-glu curonidase 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 dis ease mouse, our data provide encouragement that human trials of gene t herapy based on transferring beta-glucuronidase gene to hematopoietic cells may be efficacious.