Scientists test mRNA to fix immune cells in rare disease
NCT ID NCT05189925
Summary
This early-stage study is testing a new procedure for adults with Chronic Granulomatous Disease (CGD), a rare genetic disorder that weakens the immune system. Researchers collect a patient's white blood cells, use messenger RNA (mRNA) to temporarily correct the genetic flaw in a lab, and then infuse the corrected cells back into the patient. The main goal is to see if this one-time treatment is safe and if it can briefly help the patient's immune cells fight off germs.
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Study contacts
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Contact
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Contact
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Locations
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National Institutes of Health Clinical Center
RECRUITINGBethesda, Maryland, 20892, United States
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Conditions
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