New gene therapy offers hope for babies with 'Bubble Boy' disease
NCT ID NCT03538899
First seen Feb 15, 2026 · Last updated May 14, 2026 · Updated 16 times
Summary
This study tests a gene therapy for Artemis-deficient SCID, a severe immune disorder where babies cannot fight infections. Doctors take the child's own blood stem cells, add a working copy of the faulty gene, and return them to the body after mild chemotherapy. The goal is to build a working immune system and improve survival. The trial enrolls 24 newly diagnosed infants and follows them for 15 years.
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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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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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University of California, San Francisco (UCSF) Children's Hospital
RECRUITINGSan Francisco, California, 94143, United States
Conditions
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