Scientists test CRISPR gene editing to fight sickle cell pain

NCT ID NCT04443907

Summary

This early-stage study tested a new gene-editing treatment for people with severe sickle cell disease. Doctors collected patients' own blood stem cells, used CRISPR technology to edit a gene to boost protective fetal hemoglobin, and then transplanted the cells back after chemotherapy. The main goals were to check if the treatment was safe and if it successfully increased fetal hemoglobin levels to reduce disease complications.

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Contacts and locations

Locations

  • Memorial Sloan Kettering Cancer Ctr

    New York, New York, 10065, United States

  • St Jude Children's Research Hospital

    Memphis, Tennessee, 38105-3678, United States

  • University of Chicago

    Chicago, Illinois, 60637, United States

Conditions

Explore the condition pages connected to this study.