Busulfan pharmacokinetics toxicity and low-dose conditioning for autologous transplantation of genetically modified hematopoietic stem cells in the rhesus macaque model

摘要

ObjectiveGene 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.