Hematopoiesis has long served as a paradigm of stem cell biology and tissue homeostasis.
In the past decade, the genomics revolution has ushered in powerful new methods for …

The growing clinical success of hematopoietic stem/progenitor cell (HSPC) gene therapy
(GT) relies on the development of viral vectors as portable" Trojan horses" for safe and …

Gene therapy with LentiGlobin for sickle cell disease (bb1111, lovotibeglogene autotemcel)
consists of autologous transplantation of a patient's hematopoietic stem cells transduced …

Abstract Patients with Wiskott–Aldrich syndrome (WAS) lacking a human leukocyte antigen-
matched donor may benefit from gene therapy through the provision of gene-corrected …

Hematopoietic stem cells (HSCs) reconstitute multilineage human hematopoiesis after
clinical bone marrow (BM) transplantation and are the cells of origin of some hematological …

Gene therapy (GT) provides a potentially curative treatment option for patients with sickle
cell disease (SCD); however, the occurrence of myeloid malignancies in GT clinical trials …

Inborn errors of immunity (IEI) are diseases caused by genetic mutations that affect the
immune system's ability to fight pathogens, cope with the microbiota or regulate …

Gene transfer into autologous hematopoietic stem progenitor cells (HSPCs) has the
potential to cure monogenic inherited disorders caused by an altered development and/or …

Mutation accumulation in stem cells has been associated with cancer risk. However, the
presence of numerous mutant clones in healthy tissues has raised the question of what …

Gene editing of the erythroid-specific BCL11A enhancer in hematopoietic stem and
progenitor cells (HSPCs) from patients with sickle cell disease (SCD) induces fetal …