[HTML][HTML] Busulfan pharmacokinetics, toxicity, and low-dose conditioning for autologous transplantation of genetically modified hematopoietic stem cells in the rhesus …
EM Kang, MM Hsieh, M Metzger, A Krouse… - Experimental …, 2006 - Elsevier
OBJECTIVE: Gene transfer to hematopoietic stem cells has recently been demonstrated to
benefit a small number of patients in whom a selective advantage is conferred upon
genetically modified cells; however, in disorders where no such selective advantage is
conferred, conditioning appears necessary to allow adequate engraftment. To decrease the
toxicity profile, we sought to develop nonmyeloablative conditioning regimens and in this
work, explored the use of intravenous busulfan in a large animal model. METHODS …
benefit a small number of patients in whom a selective advantage is conferred upon
genetically modified cells; however, in disorders where no such selective advantage is
conferred, conditioning appears necessary to allow adequate engraftment. To decrease the
toxicity profile, we sought to develop nonmyeloablative conditioning regimens and in this
work, explored the use of intravenous busulfan in a large animal model. METHODS …
