First-of-its-Kind gene editing trial aims to cure devastating immune disease
NCT ID NCT06959771
Summary
This study is testing a new gene therapy for a single patient with a rare and serious inherited immune disorder called CD40L-HyperIgM syndrome. Doctors will use a precise gene-editing tool to fix the patient's own blood stem cells and immune cells, then infuse them back after a mild chemotherapy. The goal is to permanently correct the genetic error, restore a healthy immune system, and cure the disease.
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Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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National Institutes of Health Clinical Center
RECRUITINGBethesda, Maryland, 20892, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact Phone: •••-•••-•••• Email: •••••@•••••
Conditions
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