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CRISPR Technique Reawakens Dormant Genes to Treat Blood Disorders

Early tests in human blood stem cells demonstrate the method’s potential to restore hemoglobin production with fewer risks than existing therapies

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Overview

  • Scientists published a new delete-to-recruit strategy that uses CRISPR-Cas9 to remove DNA segments and bring enhancers closer to target genes.
  • The approach has successfully reactivated fetal globin in laboratory and human blood stem cells from healthy donors and patients with sickle cell disease.
  • By reviving dormant embryonic genes instead of inserting new genetic material, the method could provide a streamlined alternative for sickle cell and beta-thalassemia therapies.
  • Researchers highlight that the delete-to-recruit technique may reduce off-target risks and lower costs compared with Europe’s existing 2024-approved gene therapies.
  • Investigators suggest the enhanced control of gene-enhancer interactions could be applied to other genetic diseases where reactivating backup genes offers compensatory benefits.