Lentiviral vectors are promising tools for the genetic modification of cells in biomedical
research and gene therapy. Their use in recent clinical trials for the treatment of …

Retroviral vector–mediated gene transfer into hematopoietic stem cells provides a
potentially curative therapy for severe β-thalassemia. Lentiviral vectors based on human …

Gene therapy for the treatment of Wiskott–Aldrich syndrome (WAS) presents an alternative to
the current use of allogeneic bone marrow transplantation. We describe the development of …

Gene therapies have enormous potential to cure human disease. In recent years,
hematopoiet‐ic stem cell (HSC) gene therapy has advanced tremendously, due in part to …

Myelodysplastic syndromes (MDS) are a group of hematopoietic stem-cell disorders marked
by dysplasia that often progresses to acute myeloid leukemia (AML). The molecular analysis …

This research project stems from the successful clinical trials of gene therapy for X-linked
severe combined immunodeficiency (SCID-X1). Whilst those trials represented the first …

The safety of lentiviral (LV) vectors for gene therapy of genetic hematopoietic diseases
would be considerably enhanced by the identification of a non-viral promoter capable of …