Bubble boy disease: gene therapy trial offers hope for immune system repair
NCT ID NCT05432310
First seen Apr 10, 2026 · Last updated May 11, 2026 · Updated 5 times
Summary
This study tests a gene therapy for infants and children with a rare, life-threatening immune disorder called ADA-SCID. Doctors take the child's own blood stem cells, add a working ADA gene in the lab, and return them to the body. The goal is to restore immune function and improve survival without the need for 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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University of California, Los Angeles (UCLA)
RECRUITINGLos Angeles, California, 90095, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact Phone: •••-•••-•••• Email: •••••@•••••
Conditions
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