Adeno-associated virus 2-mediated gene transfer and functional expression of the human granulocyte-macrophage colony-stimulating factor

F. Luo, S. Z. Zhou, S. Cooper, N. C. Munshi, H. Boswell, Hal Broxmeyer, A. Srivastava

Research output: Contribution to journalArticle

11 Citations (Scopus)

Abstract

It is becoming increasingly clear that the adeno-associated virus 2 (AAV)-based vector system may prove to be useful for high-efficiency gene transfer in human cells as well as for potential gene therapy in humans. A recombinant AAV vector containing the gene for a human hematopoietic growth factor, granulocyte-macrophage colony-stimulating factor (GM-CSF), was constructed and used to infect COS-1 cells, a monkey kidney cell line. COS-1 cells infected with the recombinant virus, but not mock-infected cells, expressed high levels of the human GM-CSF gene transcripts. Furthermore, in co-cultivation experiments with the recombinant virus-infected cells, but not in those with mock-infected cells, active proliferation of a GM-CSF-dependent human megakaryocytic leukemia cell line, M07e, could be obtained in the absence of exogenously added GM-CSF. The recombinant GM-CSF protein released into the supernatant was biologically active in progenitor cell assays carried out with primary human hematopoietic cells, and this activity was specifically abrogated by treatment of the supernatant with anti-GM-CSF antibodies. This recombinant virus may be potentially useful in the management and gene therapy of a variety of malignant disorders in the human hematopoietic system.

Original languageEnglish
Pages (from-to)1261-1267
Number of pages7
JournalExperimental Hematology
Volume23
Issue number12
StatePublished - 1995

Fingerprint

Dependovirus
Granulocyte-Macrophage Colony-Stimulating Factor
Genes
COS Cells
Viruses
Genetic Therapy
Leukemia, Megakaryoblastic, Acute
Hematopoietic System
Cell Line
Haplorhini
Intercellular Signaling Peptides and Proteins
Stem Cells
Cell Proliferation
Kidney
Antibodies

Keywords

  • Adeno-associated virus
  • Gene therapy
  • Hematopoiesis
  • Recombinant DNA

ASJC Scopus subject areas

  • Cancer Research
  • Cell Biology
  • Genetics
  • Hematology
  • Oncology
  • Transplantation

Cite this

Adeno-associated virus 2-mediated gene transfer and functional expression of the human granulocyte-macrophage colony-stimulating factor. / Luo, F.; Zhou, S. Z.; Cooper, S.; Munshi, N. C.; Boswell, H.; Broxmeyer, Hal; Srivastava, A.

In: Experimental Hematology, Vol. 23, No. 12, 1995, p. 1261-1267.

Research output: Contribution to journalArticle

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