{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,5,21]],"date-time":"2026-05-21T07:44:27Z","timestamp":1779349467885,"version":"3.51.4"},"reference-count":33,"publisher":"Wiley","issue":"5","license":[{"start":{"date-parts":[[2011,6,7]],"date-time":"2011-06-07T00:00:00Z","timestamp":1307404800000},"content-version":"vor","delay-in-days":37,"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":[[2011,5]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Background<\/jats:title><jats:p>Fabry disease (FD) is a lysosomal storage disorders characterized by a deficiency of the lysosomal enzyme, \u03b1\u2010galactosidase A. This results in the accumulation of glycolipids, mainly globotriaosylceramide (GL\u20103), in the lysosomes of various organs. Although bone marrow transplantation and hematopoietic stem cell\u2010based gene therapy can offer the potential of a curative therapeutic outcome for FD, the minimum requirement of donor cells or gene\u2010corrected cells to reduce GL\u20103 levels is not known.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>Lethally\u2010irradiated FD mice were transplanted intravenously with normal bone marrow cells (Ly5.1 positive) mixed with those of FD mice (Ly5.2 positive) at various ratios to investigate the level of engraftment and enzyme activity necessary to effect a reduction in GL\u20103 storage.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>Chimerism of whole white blood cells of recipients' peripheral blood remained stable at 8 weeks after transplantation, and chimerism of granulocytes, monocytes, B cells and T cells was equal to that of white blood cells. GL\u20103 levels were significantly reduced in the lung and heart of animals with a 30% and 50% chimera, respectively. The extent of reduction in these mice was almost identical to that with 100% chimera.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusions<\/jats:title><jats:p>In FD mice, reconstitution with 100% donor cells is not required to obtain a therapeutic effect following bone marrow transplantation. These results suggest that a 30% gene correction might be sufficient to reverse disease manifestations in FD. Copyright \u00a9 2011 John Wiley &amp; Sons, Ltd.<\/jats:p><\/jats:sec>","DOI":"10.1002\/jgm.1566","type":"journal-article","created":{"date-parts":[[2011,4,26]],"date-time":"2011-04-26T02:13:34Z","timestamp":1303784014000},"page":"262-268","source":"Crossref","is-referenced-by-count":10,"title":["Minimum requirement of donor cells to reduce the glycolipid storage following bone marrow transplantation in a murine model of Fabry 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