Pioneering gene therapy trials have shown that the genetic engineering of haematopoietic
stem and progenitor cells can be an alternative to allogeneic transplantation in the treatment …

The ability of the bone marrow (BM) to generate copious amounts of blood cells required on
a daily basis depends on a highly orchestrated process of proliferation and differentiation of …

Genome editing is a powerful new tool for making precise alterations to an organism's
genetic material. Recent scientific advances have made genome editing more efficient …

Genetic engineering of hematopoiesis: current stage of clinical translation and future
perspectives | EMBO Molecular Medicine Skip to Article Content Skip to Article Information All …

Innate immune factors may restrict hematopoietic stem cell (HSC) genetic engineering and
contribute to broad individual variability in gene therapy outcomes. Here, we show that …

Lentiviral vectors (LVs) are increasingly employed in gene and cell therapy. Standard
laboratory production of LVs is not easily scalable, and research-grade LVs often contain …

Clinical application of lentiviral vector (LV)‐based hematopoietic stem and progenitor cells
(HSPC) gene therapy is rapidly becoming a reality. Nevertheless, LV‐mediated signaling …

Genome engineering using CRISPR/Cas9 technology enables simple, efficient and precise
genomic modifications in human cells. Conventional immortalized cell lines can be easily …

Transduction of primary human natural killer (NK) cells with lentiviral vectors has historically
been challenging. We sought to evaluate multiple parameters to optimize lentiviral …

Viral vectors play a pivotal role in the field of gene therapy, with several related drugs having
already gained clinical approval from the EMA and FDA. However, numerous viral gene …