Due to their efficient recognition and lysis of malignant cells, natural killer (NK) cells are
considered as specialized immune cells that can be genetically modified to obtain capable …

Chimeric antigen receptors (CARs) are fusion proteins with an extracellular antigen
recognition domain and numerous intracellular signaling domains that have been …

The use of allogeneic hematopoietic stem cells (HSCs) to treat genetic blood cell diseases
has become a clinical standard but is limited by the availability of suitable matched donors …

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 …

Ex vivo gene therapy based on CD34+ hematopoietic stem cells (HSCs) has shown
promising results in clinical trials, but genetic engineering to high levels and in large scale …

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 …

Recently, gene therapy clinical trials have been successfully applied to
hemoglobinopathies, such as sickle cell disease (SCD) and β-thalassemia. Among the great …

Rapamycin and its derivatives are specific inhibitors of mammalian target of rapamycin
(mTOR) kinase and, as a result, are well-established immunosuppressants and …

Gene therapy currently in development for hemoglobinopathies utilizes ex vivo lentiviral
transduction of CD34+ hematopoietic stem and progenitor cells (HSPCs). A small-molecule …