In vivo selection of retrovirally transduced hematopoietic stem cells (original) (raw)

Gene therapy presents a viable option for diseases responsive to allogeneic bone marrow transplantation, particularly those stemming from single gene defects. However, gene transfer efficiency remains low in clinical applications, necessitating strategies to enhance the number of modified hematopoietic stem cells (HSCs). This paper discusses the development of retroviral vectors aimed at improving gene transfer efficiency. It specifically explores in vivo selection mechanisms that utilize selectable markers to enrich transduced HSCs, highlighting the challenges encountered, such as the lack of effective systems for repopulating stem cells. The findings demonstrate the possibilities and limitations of current strategies in achieving stable enrichment of gene-modified hematopoietic cells.