A groundbreaking advancement in hematopoietic stem cell transplantation (HSCT) using Prime Editing technology was recently presented at the 66th Annual ASH Congress, offering a safer and more precise alternative to conventional conditioning methods like chemotherapy and radiation.

Conventional approaches, while effective, often lead to severe multi-organ damage and increased treatment-associated risks. In this innovative approach, researchers utilized Prime Editing to introduce a protective mutation in the KIT gene of CD34+ stem cells, shielding them from depletion by CD117 antibodies while preserving their functional integrity.

Clinical-scale results demonstrated that 73% of CD34+ cells were successfully edited, with 50% containing biallelic edits, while retaining full multipotency and viability.

When tested in immunodeficient mice, these shielded cells showed successful engraftment without adverse effects, highlighting the potential of Prime Editing to revolutionize HSCT by reducing toxic conditioning side effects and enhancing treatment precision for blood disorders and genetic diseases.

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