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Gene Transfer into Human Hematopoietic Stem Cells
Published in Richard K. Burt, Alberto M. Marmont, Stem Cell Therapy for Autoimmune Disease, 2019
Serguei Kisselev, Tatiana Seregina, Richard K. Burt, Charles J. Link
Some potential approaches may override this problem. One is to use highly purified primitive progenitor cells in gene transfer clinical trials. Another approach is to use pseudotyped and/or targeted vectors that possess an intracellular transport ability independent of RAM-1 expression. There were three main types of vector systems developed based on the pseudotyped MoMuLV platform. Each vector allows more effective gene transfer into various types of human cells compared with vectors packaged in the amphotropic envelopes. Investigators demonstrated that gibbon ape leukemia virus (GALV) envelope can be successfully used to pseudotype murine retroviruses allowing improved gene transfer into higher primate and human cells.57 The cellular receptor for the GALV envelope is an inducible sodium phosphate importer (GALVR-1) distinct from RAM-1.58 GALV-pseudotyped retroviruses significantly improve gene transfer into human HSC capable of repopulating NOD-SCID mice suggesting that GALV-pseudotyped retroviruses can successfully transduce populations of very primitive progenitor cells.59
Novel BCMA-OR-CD38 tandem-dual chimeric antigen receptor T cells robustly control multiple myeloma
Published in OncoImmunology, 2021
Yaru Feng, Xiuying Liu, Xiaorui Li, Yating Zhou, Zhiru Song, Jing Zhang, Bingjie Shi, Jianxun Wang
K562 cells were obtained from the Cell Resource Center, Peking Union Medical College (Beijing, China). Raji cells are a CD38+ human B lymphocyte cell line that was obtained from the American Type Culture Collection (ATCC, United States). BCMA+ GFP+-K562 cells were a generous gift from Dr. Wu (China Agricultural University) received in 2018. GFP-Luciferase RPMI8226 cells (RPMI-Luc) were a CD38+ and BCMA+ myeloma cell line that were obtained from the ATCC. The PG13 gibbon ape leukemia virus packaging cell line and the human ecotropic packaging cell line Phoenix-ECO were obtained from ATCC. K562, Raji, and RPMI cells were maintained in RPMI-1640 medium (Gibco, United States) supplemented with 10% fetal bovine serum (FBS) (Gibco, United States), with 1% penicillin-streptomycin (P/S) solution (Gibco, United States). PG13 and Phoenix ECO cells were cultured in DMEM (Gibco, United States) supplemented with 10% FBS and 1% P/S. All cells were cultured in an incubator (Thermo Fisher, United States) at 37°C and 5% CO2. All cell lines were tested negative for the mycoplasma contamination and cell-surface markers for these cell lines were validated by flow cytometry.