Scientists test mRNA 'Repair' for rare immune disease

NCT ID NCT05189925

Summary

This early-stage study is testing a one-time treatment for Chronic Granulomatous Disease (CGD), a rare genetic disorder where the immune system cannot fight infections properly. Researchers will collect a patient's white blood cells, use messenger RNA (mRNA) to temporarily fix the genetic error in a lab, and then infuse the corrected cells back into the patient. The main goals are to see if this procedure is safe and to find the best dose, with the hope of giving patients's immune cells a temporary boost to fight germs.

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Contacts and locations

Study contacts

  • Contact

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

  • Contact

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

Locations

  • National Institutes of Health Clinical Center

    RECRUITING

    Bethesda, Maryland, 20892, United States

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

Conditions

Explore the condition pages connected to this study.