The efficacy of adoptive T-cell therapy as treatment for malignancies may be enhanced by
genetic modification of infused cells. However, oncogenic events due to vector/transgene …

Chronic granulomatous disease (CGD) is a congenital immune deficiency that is a
promising therapeutic target for gene replacement into haematopoietic stem cells (HSCs) …

The clonal dynamics following hematopoietic stem progenitor cell (HSPC) transplantation
with busulfan conditioning are of great interest to the development of HSPC gene therapies …

Although gene transfer to hematopoietic stem cells (HSCs) has shown therapeutic efficacy in
recent trials for several individuals with inherited disorders, transduction incompleteness of …

Background Human monocytes play an important role in mediating human
immunodeficiency virus type 1 (HIV‐1) infection of the central nervous system (CNS), and …

Efficient selection of gene-modified cells is required for a number of potential gene therapy
applications, as well as molecular biology studies. Ideally, a clinical selection regimen would …

This study assessed the feasibility of using an ex vivo stem cell antigen-1–positive (Sca-1+)
cell–based systemic fibroblast growth factor-2 (FGF-2) gene therapy to promote endosteal …

The incorporation of an endogenous safety switch represents a rational strategy for the
control of toxicities following the administration of adoptive T cell therapies. An ideal safety …

To improve maintenance and gene transfer of human lymphoid progenitors for clinical use in
gene therapy of adenosine deaminase (ADA)-deficient SCID we investigated several gene …

Background Tumor cell contamination of the apheresis in multiple myeloma is likely to affect
disease-free and overall survival after autografting. Objective To purge myeloma aphereses …