{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2023,10,19]],"date-time":"2023-10-19T05:17:25Z","timestamp":1697692645615},"reference-count":21,"publisher":"Wiley","issue":"4","license":[{"start":{"date-parts":[[2004,2,10]],"date-time":"2004-02-10T00:00:00Z","timestamp":1076371200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["The Journal of Gene Medicine"],"published-print":{"date-parts":[[2004,4]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Background<\/jats:title><jats:p>Gene transfer efficiency into primitive hematopoietic cells may be limited by their expression of surface receptors allowing vector entry. Vectors pseudotyped with the vesicular stomatitis virus (VSV\u2010G) envelope do not need receptors to enter cells, and therefore may provide superior transduction efficiency.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>Using a competitive repopulation model in the rhesus macaque, we examined <jats:italic>in vivo<\/jats:italic> gene marking levels of blood cells transduced with two vectors: (i) a VSV\u2010G pseudotyped retrovirus and (ii) a conventional amphotropic retrovirus. The VSV\u2010G vector, containing the human glucose\u20106\u2010phosphate dehydrogenase (G6PD) gene, was constructed for treatment of severe hemolytic anemia caused by G6PD deficiency. Three myeloablated animals were transplanted with peripheral blood CD34+ cells, half of which were transduced with the VSV\u2010G vector and the other half with the amphotropic vector.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>In all animals post\u2010transplantation, levels of <jats:italic>in vivo<\/jats:italic> marking in circulating granulocytes and mononuclear cells were similar: 1% or less with both vectors. In one animal, the human G6PD enzyme transferred by the VSV\u2010G vector was expressed in erythrocytes, early after transplantation, at a level of 45% of the endogenous rhesus G6PD protein.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusions<\/jats:title><jats:p>In a clinically relevant animal model, we found similar <jats:italic>in vivo<\/jats:italic> marking with a VSV\u2010G pseudotyped and a standard amphotropic oncoretroviral vector. Amphotropic receptor expression may not be a limiting factor in transduction efficiency, but VSV\u2010G pseudotypes possess other practical advantages that may make them advantageous for clinical use. 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