MISTRG mice support engraftment and assessment of nonhuman primate hematopoietic stem and progenitor cells

被引:15
|
作者
Radtke, Stefan [1 ]
Chan, Yan-Yi [1 ]
Sippel, Trisha R. [2 ]
Kiem, Hans-Peter [1 ,3 ,4 ]
Rongvaux, Anthony [2 ,5 ]
机构
[1] Fred Hutchinson Canc Res Ctr, Div Clin Res, Program Cell & Gene Therapy, 1124 Columbia St, Seattle, WA 98104 USA
[2] Fred Hutchinson Canc Res Ctr, Div Clin Res, Program Immunol, 1124 Columbia St, Seattle, WA 98104 USA
[3] Univ Washington, Sch Med, Dept Med, Seattle, WA 98195 USA
[4] Univ Washington, Sch Med, Dept Pathol, Seattle, WA 98195 USA
[5] Univ Washington, Sch Med, Dept Immunol, Seattle, WA USA
基金
美国国家卫生研究院;
关键词
TRANSPLANTATION; POPULATIONS; IL-3;
D O I
10.1016/j.exphem.2018.12.003
中图分类号
R5 [内科学];
学科分类号
1002 ; 100201 ;
摘要
Preclinical feasibility, safety, and efficacy testing of hematopoietic stem cell (HSC)-mediated gene therapy approaches is commonly performed in large-animal models such as nonhuman primates (NHPs). Here, we wished to determine whether mouse models would allow engraftment of NHP HSPCs, which would enable more facile and less costly evaluation of promising strategies. In this study, we comprehensively tested two mouse strains for the engraftment of NHP CD34(+) hematopoietic stem and progenitor cells (HSPCs). No engraftment of NHP HSPCs was observed in NSG mice, whereas the gene-humanized MISTRG model did demonstrate dose-dependent multilineage engraftment of NHP cells in the peripheral blood, bone marrow, spleen, and thymus. Most importantly, and closely mimicking the hematopoietic recovery of autologous stem cell transplantations in the NHP, only HSC-enriched CD34(+)CD90(+)CD45RA(-) cell fractions engrafted and reconstituted the bone marrow stem cell niche in MISTRG mice. In summary, we here report the first "monkeynized" mouse xenograft model that closely recapitulates the autologous hematopoietic reconstitution in the NHP stem and progenitor cell transplantation and gene therapy model. The availability of this model has the potential to pre-evaluate novel HSC-mediated gene therapy approaches, inform studies in the NHP, and improve the overall outcome of large-animal experiments. (C) 2019 Published by Elsevier Inc. on behalf of ISEH - Society for Hematology and Stem Cells.
引用
收藏
页码:31 / 41
页数:11
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