Abstract
Two-gene vectors with positive or positive-negative drug-selectable markers enable the expansion or elimination of gentetically modified cells in vivo. We have established a bicistronic retroviral vector system which utilizes an internal ribosome entry site (IRES) to co-express two independent genes with high efficiency. As a positive-negative (suicide) marker, Herpes simplex virus thymidine kinase was co-expressed with the human multidrug resistance gene, MDR1. Using this vector, almost all the MDR1-transduced cells showed hypersensitivity to a nucleoside analog, ganciclovir. As a dominant selectable marker, the MDR1 gene was co-expressed with α-galactosidase A for the model of gene therapy of Fabry disease. Vincristine selection efficiently enhanced the population of transduced cells expressing the second non-selectable genes. These drug-selectable retroviral vectors could be applicable to the therapy of many diseases.
| Original language | English |
|---|---|
| Pages (from-to) | 552-556 |
| Number of pages | 5 |
| Journal | Leukemia |
| Volume | 11 |
| Issue number | SUPPL. 3 |
| Publication status | Published - 1997 |
| Externally published | Yes |
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ASJC Scopus subject areas
- Hematology
- Cancer Research
Cite this
In vivo drug-selectable markers in gene therapy. / Sugimoto, Yoshikazu; Tsuruo, T.
In: Leukemia, Vol. 11, No. SUPPL. 3, 1997, p. 552-556.Research output: Contribution to journal › Article
}
TY - JOUR
T1 - In vivo drug-selectable markers in gene therapy
AU - Sugimoto, Yoshikazu
AU - Tsuruo, T.
PY - 1997
Y1 - 1997
N2 - Two-gene vectors with positive or positive-negative drug-selectable markers enable the expansion or elimination of gentetically modified cells in vivo. We have established a bicistronic retroviral vector system which utilizes an internal ribosome entry site (IRES) to co-express two independent genes with high efficiency. As a positive-negative (suicide) marker, Herpes simplex virus thymidine kinase was co-expressed with the human multidrug resistance gene, MDR1. Using this vector, almost all the MDR1-transduced cells showed hypersensitivity to a nucleoside analog, ganciclovir. As a dominant selectable marker, the MDR1 gene was co-expressed with α-galactosidase A for the model of gene therapy of Fabry disease. Vincristine selection efficiently enhanced the population of transduced cells expressing the second non-selectable genes. These drug-selectable retroviral vectors could be applicable to the therapy of many diseases.
AB - Two-gene vectors with positive or positive-negative drug-selectable markers enable the expansion or elimination of gentetically modified cells in vivo. We have established a bicistronic retroviral vector system which utilizes an internal ribosome entry site (IRES) to co-express two independent genes with high efficiency. As a positive-negative (suicide) marker, Herpes simplex virus thymidine kinase was co-expressed with the human multidrug resistance gene, MDR1. Using this vector, almost all the MDR1-transduced cells showed hypersensitivity to a nucleoside analog, ganciclovir. As a dominant selectable marker, the MDR1 gene was co-expressed with α-galactosidase A for the model of gene therapy of Fabry disease. Vincristine selection efficiently enhanced the population of transduced cells expressing the second non-selectable genes. These drug-selectable retroviral vectors could be applicable to the therapy of many diseases.
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UR - http://www.scopus.com/inward/citedby.url?scp=0030795066&partnerID=8YFLogxK
M3 - Article
C2 - 9209454
AN - SCOPUS:0030795066
VL - 11
SP - 552
EP - 556
JO - Leukemia
JF - Leukemia
SN - 0887-6924
IS - SUPPL. 3
ER -