The function of this core unit is to assist investigators of this PPG in the in vivo functional evaluation of human cells transduced by their vectors. This will be achieved by using transplantations of transduced human hemopoietic cells into a receptive xenogeneic host (NOD/SCID or NOD/SCID/beta2/mug-/-). The unit will generate and maintain sufficient numbers of NOD/SCID or NOD/SCID/beta2/mug-/- mice, to accommodate the needs of investigators of this PPG, and will be responsible for the mechanics of transplantation (i.e., irradiation, i.v., or intramarrow injection of donor cells). In addition, the unit will undertake the sampling and assist, at least in part, in the post-transplant evaluation required to assess the fate of the transplanted human cells.
| Constantinou, Varnavas C; Bouinta, Asimina; Karponi, Garyfalia et al. (2017) Poor stem cell harvest may not always be related to poor mobilization: lessons gained from a mobilization study in patients with ?-thalassemia major. Transfusion 57:1031-1039 |
| Gori, Jennifer L; Butler, Jason M; Kunar, Balvir et al. (2017) Endothelial Cells Promote Expansion of Long-Term Engrafting Marrow Hematopoietic Stem and Progenitor Cells in Primates. Stem Cells Transl Med 6:864-876 |
| Psatha, Nikoletta; Karponi, Garyfalia; Yannaki, Evangelia (2016) Optimizing autologous cell grafts to improve stem cell gene therapy. Exp Hematol 44:528-39 |
| Li, Li B; Ma, Chao; Awong, Geneve et al. (2016) Silent IL2RG Gene Editing in Human Pluripotent Stem Cells. Mol Ther 24:582-91 |
| Karponi, Garyfalia; Psatha, Nikoletta; Lederer, Carsten Werner et al. (2015) Plerixafor+G-CSF-mobilized CD34+ cells represent an optimal graft source for thalassemia gene therapy. Blood 126:616-9 |
| Vierstra, Jeff; Reik, Andreas; Chang, Kai-Hsin et al. (2015) Functional footprinting of regulatory DNA. Nat Methods 12:927-30 |
| Qi, Heyuan; Liu, Mingdong; Emery, David W et al. (2015) Functional validation of a constitutive autonomous silencer element. PLoS One 10:e0124588 |
| Liu, Mingdong; Maurano, Matthew T; Wang, Hao et al. (2015) Genomic discovery of potent chromatin insulators for human gene therapy. Nat Biotechnol 33:198-203 |
| Polak, Paz; Karli?, Rosa; Koren, Amnon et al. (2015) Cell-of-origin chromatin organization shapes the mutational landscape of cancer. Nature 518:360-364 |
| Chandrasekaran, Devikha; Nakamoto, Betty; Watts, Korashon L et al. (2014) Modeling promising nonmyeloablative conditioning regimens in nonhuman primates. Hum Gene Ther 25:1013-22 |
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