CRISPR-Edited Stem Cells Could Make Blood-Cancer Therapy Safer

On 25th September 2026, researchers reported results from a phase 1/2 clinical trial in which donor blood stem cells were edited using CRISPR-Cas9 to remove the protein CD33, potentially allowing aggressive blood cancers to be targeted while sparing healthy blood-forming cells.

How Does the Technology Work?

  • CD33 Targeting Challenge: In diseases such as acute myeloid leukaemia (AML) and myelodysplastic syndrome (MDS), CD33 is present on cancer cells as well as healthy myeloid cells. Thus, CD33-targeted therapies may damage healthy blood cells along with cancer cells.
  • CRISPR-Cas9 Gene Editing: Researchers genetically modified donor haematopoietic stem cells before transplantation so that the healthy ....
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