Gene therapy could replace bone marrow transplants for rare immune disease
NCT ID NCT06736080
First seen May 27, 2026 · Last updated May 29, 2026 · Updated 1 time
Summary
This trial tests a gene therapy for FHL3, a rare immune disorder caused by a gene mutation. Instead of a donor stem cell transplant, patients receive their own stem cells that have been corrected with a healthy gene. The goal is to restore immune function while avoiding transplant complications like graft-versus-host disease. Up to 5 patients aged 3 months to 45 years will be enrolled.
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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 Biotherapy, Hopital Necker Enfants Malades
Paris, 75015, France
Contact Phone: •••-•••-•••• Email: •••••@•••••
Conditions
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