In vivo drug-selectable markers in gene therapy

Research output: Contribution to journalArticle

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 languageEnglish
Pages (from-to)552-556
Number of pages5
JournalLeukemia
Volume11
Issue numberSUPPL. 3
Publication statusPublished - 1997
Externally publishedYes

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Genetic Therapy
Pharmaceutical Preparations
Genes
Galactosidases
MDR Genes
Fabry Disease
Ganciclovir
Thymidine Kinase
Vincristine
Simplexvirus
Nucleosides
Suicide
Hypersensitivity
Population
Therapeutics

ASJC Scopus subject areas

  • Hematology
  • Cancer Research

Cite this

Sugimoto, Y., & Tsuruo, T. (1997). In vivo drug-selectable markers in gene therapy. Leukemia, 11(SUPPL. 3), 552-556.

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 journalArticle

Sugimoto, Y & Tsuruo, T 1997, 'In vivo drug-selectable markers in gene therapy', Leukemia, vol. 11, no. SUPPL. 3, pp. 552-556.
Sugimoto, Yoshikazu ; Tsuruo, T. / In vivo drug-selectable markers in gene therapy. In: Leukemia. 1997 ; Vol. 11, No. SUPPL. 3. pp. 552-556.
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