One-Time gene therapy aims to cure 'Bubble Boy' disease
NCT ID NCT05071222
Summary
This early-stage trial is testing a one-time gene therapy for babies with a rare and severe form of immune deficiency called Artemis SCID. Doctors take a patient's own blood stem cells, use a virus to insert a corrected gene in the lab, and then transplant the cells back. The goal is to see if this can safely rebuild the immune system, potentially curing the disease without needing a donor.
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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
Explore the condition pages connected to this study.