Background Chimeric antigen receptor (CAR) T-cell therapies have demonstrated
transformational outcomes in the treatment of B-cell malignancies, but their widespread use …

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The tremendous evolution of genome-editing tools in the last two decades has provided
innovative and effective approaches for gene therapy of congenital and acquired diseases …

Chimeric antigen receptor (CAR) T-cell therapies have demonstrated transformative efficacy
in treating B-cell malignancies. However, high costs and manufacturing complexities hinder …

Human CD34+ hematopoietic stem and progenitor cells (HSPCs) are a standard source of
cells for clinical HSC transplantations as well as experimental xenotransplantation to …

Gene therapy is a relatively novel field that amounts to around four decades of continuous
growth with its good and bad moments. Currently, the field has entered the clinical arena …

Drug delivery key research aim is to support patients by designing clinically effective
formulations. Drug delivery systems can enhance the treatment of a variety of diseases …

Abstract Hematopoietic Stem Cell (HSC) gene therapy is a promising route to curing patients
with a variety of hematologic diseases and disorders. HSC gene therapy is currently …

IT HAS BECOME A NOW long-standing tradition that we launch a Human Gene Therapy
special issue in conjunction with the annual congress of the European Society of Gene and …

Hematopoietic stem cell (HSC) gene therapy is currently performed on CD34+
hematopoietic stem and progenitor cells containing less than 1% true HSCs and requiring a …