Scientists test 'Base Editing' to fix faulty immune cells

NCT ID NCT06325709

Summary

This study is testing a new gene-editing treatment for adults with a rare, inherited immune disorder called X-linked chronic granulomatous disease (X-CGD). Doctors will collect a patient's own blood stem cells, use a precise gene-editing tool called 'base editing' to try to fix the genetic error, and then return the cells to the patient after chemotherapy. The goal is to see if this one-time treatment can restore proper immune function and reduce severe infections.

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Contacts and locations

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

Locations

  • National Institutes of Health Clinical Center

    RECRUITING

    Bethesda, Maryland, 20892, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact Phone: •••-•••-•••• Email: •••••@•••••

Conditions

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