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.
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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)
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Chemical References |
- Antigens, CD34
- Glucuronidase
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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
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