2013年11月15日 · We have developed a scalable process capable of deriving >300 million gene-modified CD34+ HSPC. This process supports high levels of ZFN-driven genome editing, is well tolerated, and causes no discernable defect in the hematopoietic potential of these cells to develop into multiple cell lineages, with high gene editing levels maintained in the …

A. Quantile-quantile plot comparing differentially expressed genes between GPI-80 + and GPI-80 − HSPC is shown. B. Bar graphs showing the relative expression of known transcription factors required for HSC development or maintenance.C. Bar graphs showing the relative expression of transcription factors responsible for lineage differentiation.

Original Article PGE2 and Poloxamer Synperonic F108 Enhance Transduction of Human HSPCs with a b-Globin Lentiviral Vector Katelyn E. Masiuk, 1Ruixue Zhang, Kyle Osborne, Roger P. Hollis,1 Beatriz Campo-Fernandez,1 and Donald B. Kohn1 ,23 1Department of Microbiology, Immunology & Molecular Genetics, University of California, Los Angeles, Los Angeles, CA …

2023年5月4日 · Naldini et al. review the state-of-the-art of hematopoietic stem/progenitor cell (HSPC) gene therapy from gene transfer to the cutting-edge gene editing technologies. Lessons learned about opportunities and hurdles of HSPC biology for genetic engineering, and vice versa, should instruct wise choices of gene therapy modalities for each therapeutic application.

2019年6月14日 · Lentiviral vector (LV)-based hematopoietic stem and progenitor cell (HSPC) gene therapy is becoming a promising alternative to allogeneic stem cell transplantation for curing genetic diseases. Clinical trials are currently underway to treat sickle cell disease using LVs expressing designed anti-sickling globin genes. However, because of the large size and …

Isolation and growth properties of human CD34 + and CD34 + /CD38 − cells. (A): Flow cytometry of CD34-enriched cells showing gating strategy used to define CD34 + /CD38 + cells (region P5) and CD34 + /CD38 − cells (region P3). (B): Cell expansion from CD34 + and CD34 + /CD38 − cells from cord blood. Cells were cocultured with irradiated MS5 stromal cells in long-term …

2017年12月28日 · Author summary Hematopoietic Stem/Progenitor Cell (HSPC) based gene therapy can be used to treat many infectious and genetic diseases. Here, we used an HSPC-based approach to redirect and enhance host immunity against HIV-1. We engineered HSPCs to carry chimeric antigen receptor (CAR) genes that detect and destroy HIV-infected cells. CAR …

11. Cell and Developmental Biology, Northwestern University, Feinberg School of Medicine, Chicago, IL 60611, USA. 12. Department of Paediatrics, Faculty of Medicine, Dentistry and Health Sciences, University of

2024年2月7日 · Hematopoietic stem/progenitor cell (HSPC)-based anti-HIV-1 gene therapy holds great promise to eradicate HIV-1 or to provide long-term remission through a continuous supply of anti-HIV-1 gene-modified cells without ongoing antiretroviral therapy. However, achieving sufficient engraftment levels of a …

Pluripotent stem cells (PSCs) may provide a potential source of haematopoietic stem/progenitor cells (HSPCs) for transplantation; however, unknown molecular barriers prevent the self-renewal of PSC-HSPCs. Using two-step differentiation, human embryonic stem cells (hESCs) differentiated in vitro into …