{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,1,9]],"date-time":"2026-01-09T02:01:32Z","timestamp":1767924092460,"version":"3.49.0"},"reference-count":57,"publisher":"Wiley","issue":"7","license":[{"start":{"date-parts":[[2005,2,11]],"date-time":"2005-02-11T00:00:00Z","timestamp":1108080000000},"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":[[2005,7]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Background<\/jats:title><jats:p>Gaucher disease is a lysosomal storage disorder resulting from a deficiency of glucocerebrosidase (GC). Recently, lentivirus vectors have been developed for efficient gene transfer into hematopoietic stem cells (HSCs). A recombinant lentivirus vector was used to evaluate the transduction of the human GC gene into murine bone\u2010marrow\u2010derived HSCs and its expression in their progeny.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>Murine HSCs were transduced with lentivirus vector (lenti\u2010EF\u2010GC; MOI = 10\u2013100). We transplanted female wild\u2010type C57BL\/6J mice with genetically modified male HSCs via the tail vein.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>We show that intravenous transplantation of transduced HSCs has therapeutic potential. Enzyme activity was increased two\u2010 to three\u2010fold in various tissues, especially in the hematopoietic system. Numerous transplanted HSCs survived for 6 months and were shown by PCR to contain the provirus genes; the Y chromosome was identified by FISH analysis in the cells of female mouse recipients.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusions<\/jats:title><jats:p>The recombinant lentiviral vector transduces HSCs that are capable of long\u2010term gene expression <jats:italic>in vivo<\/jats:italic>. This approach is potentially useful for the treatment of patents with Gaucher disease and other lysosomal storage disorders. Copyright \u00a9 2005 John Wiley &amp; Sons, Ltd.<\/jats:p><\/jats:sec>","DOI":"10.1002\/jgm.732","type":"journal-article","created":{"date-parts":[[2005,2,11]],"date-time":"2005-02-11T14:18:36Z","timestamp":1108131516000},"page":"878-887","source":"Crossref","is-referenced-by-count":17,"title":["Long\u2010term expression of the human glucocerebrosidase gene <i>in vivo<\/i> after transplantation of bone\u2010marrow\u2010derived cells transformed with a lentivirus vector"],"prefix":"10.1002","volume":"7","author":[{"given":"Eun Young","family":"Kim","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Young 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