Efficient and persistent expression of beta-glucuronidase gene in CD34+ cells from human umbilical cord blood by retroviral vector.

Abstract

We succeeded in efficiently transferring the beta-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 beta-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 beta-glucuronidase gene to hematopoietic cells may be efficacious.

• Authors: T Ohashi, S Iizuka, W S Sly, K Machiki, Y Eto
• Journal: European journal of haematology (Eur J Haematol) Vol. 61 Issue 4 Pg. 235-9 (Oct 1998) ISSN: 0902-4441 [Print] England
• PMID: 9820629 (Publication Type: Journal Article)

Chemical References

• Antigens, CD34
• Glucuronidase

Topics

• Animals
• Antigens, CD34
• Fetal Blood (cytology)
• Gene Expression
• Gene Transfer Techniques
• Genetic Vectors
• Glucuronidase (genetics)
• Hematopoietic Stem Cell Transplantation
• Hematopoietic Stem Cells (physiology)
• Humans
• Mice
• Retroviridae