One-Time gene fix could cure babies with 'Bubble Boy' disease
NCT ID NCT05071222
First seen Mar 31, 2026 · Last updated May 06, 2026 · Updated 4 times
Summary
This study tests a one-time gene therapy for children with Artemis SCID, a severe immune disorder that leaves them vulnerable to infections. Doctors take the child's own blood stem cells, fix the faulty gene in a lab, and return them to the body. The goal is to restore a working immune system without the need for a donor transplant or lifelong medication.
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Department of Pediatric Immunology, Hematology and Rheumatology UIHR, Necker-Enfants Malades Hospital
RECRUITINGParis, 75015, France
Contact Email: •••••@•••••
Conditions
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