New Gene Therapy Targets Blood Stem Cells Directly, Potentially Eliminating Need for Transplants

New Gene Therapy Targets Blood Stem Cells Directly, Potentially Eliminating Need for Transplants
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Researchers at the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan have identified a critical early postnatal window that allows direct gene therapy targeting of circulating blood stem cells. Published in Nature, the study reveals that shortly after birth, blood stem and progenitor cells (HSPCs) circulate in higher numbers, enabling effective in vivo gene transfer via lentiviral vectors through a simple injection.

This approach contrasts with traditional ex vivo gene therapy, which requires harvesting stem cells, modifying them in the lab, and reinfusing after chemotherapy—a complex and invasive process. The new method showed therapeutic benefits in mouse models of ADA-SCID, osteopetrosis, and Fanconi anemia, with corrected stem cells repopulating the blood system and improving survival.

The team also used mobilizing drugs to increase circulating stem cells, extending the treatment window. Importantly, circulating HSPCs were detected in human newborns, suggesting potential clinical application.

While less efficient than current ex vivo methods, this in vivo strategy could offer a less invasive treatment for severe genetic blood disorders in infants, marking a significant step toward new gene therapy options.