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Updated: Jul 2, 2025
Busulfan as a Myelosuppressive Agent for Generating Stable High-level Bone Marrow Chimerism in Mice
Published on: April 1, 2015
The Effects of Busulfan on Xenogeneic Transplantation of Human Peripheral Blood Mononuclear Cells in Recipient Mice
Inseon Bak1, Myeongjin Choi2, Eunhye Yu3
1GHBIO Inc. (Genes & Health Biotechnology), Yuseong-gu, Daejeon, Republic of Korea; Immunology and Immunopharmacology Laboratory, College of Pharmacy, Chungnam National University, Yuseong-gu, Daejeon, Republic of Korea.
Background:
Humanized mouse models with engraftment of human peripheral blood mononuclear cells (PBMCs) or hematopoietic stem cells (HSCs) are effective tools for the study of human immunity. Busulfan has been used as a substitute for irradiation in human hematopoietic stem cell (HSC) transplantation models, but it has not been tested in human peripheral blood mononuclear cell (PBMC) transplantation models.
Methods:
This study evaluated PBMC engraftment using cytometry and enzyme-linked immunosorbent assay (ELISA) in female NOD.CB17/Prkdcscid/JKrb/ IL2 receptor γ-/- (NIG) mice treated with busulfan.
Results:
In this model, the percentage of human CD3+ T cell engraftment in the blood was 28.2%, with dominant infiltration of CD8+ cells in the spleen 3 weeks post PBMC transplantation. Production of human cytokines, including Interleukin (IL)-12p70, IL-4, IL-5, IFN-γ, IL-6, IL-8, IL-22, Tumor Necrosis Factor alpha, and IL-10, was determined in mice treated with busulfan.
Conclusions:
Our findings demonstrate that busulfan treatment is a beneficial alternative for simple and efficient PBMC engraftment in a rodent model, possibly helping to evaluate human immunity in preclinical studies.
