PMID: 11166455Feb 13, 2001Paper

Analysis of gene transfer efficiency of retrovirus producer cell transplantation for in situ gene transfer to hematopoietic cells

Experimental Hematology
G C NewboundB A Bunnell

Abstract

The aim of this study was to assess the gene transfer efficiency of an in situ administration protocol for hematopoietic stem/progenitor cells in the rhesus macaque (Macaca mulatta) animal model. Moloney murine leukemia virus amphotropic vector producer cells (1--2 x 10(8) cells/animal) were transplanted into the femoral bone marrow cavities of six macaques. To determine if the levels of gene transfer could be increased, a second injection at the same dose of producer cells was performed into the iliac crest in three of the six macaques. We demonstrated that 0.02-0.1% of peripheral blood mononuclear cells contained the vector transgene for up to 12 months following the initial administration of producer cells. Hematopoietic progenitor cell assays indicated that the neomycin phosphotransferase gene was detected in 10--30% of progenitor cell colonies. A humoral immune response directed toward viral particles was demonstrated in all animals. Additionally, we demonstrated that an increase in the levels of transduced cells, up to 1% of circulating peripheral blood mononuclear cells and granulocytes, contain the transgene following producer cell readministration. These data demonstrate the successful in situ gene transfer to hematopo...Continue Reading

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Citations

Aug 9, 2006·Molecular Therapy : the Journal of the American Society of Gene Therapy·D Nicole WorshamDao Pan
Sep 1, 2004·Molecular Therapy : the Journal of the American Society of Gene Therapy·Peter A HornHans-Peter Kiem
Mar 26, 2008·Gene Therapy·Fabio CandottiJ M Puck

Related Concepts

Antibodies, Viral
Marrow
Trochanter
Flow Cytometry
Shuttle Vectors
Colony-Forming Units, Hematopoietic
Luminescent Proteins
Macaca mulatta
Moloney Leukemia Virus
RNA

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