Bubble boy disease gets a gene fix: new trial offers hope
NCT ID NCT06851767
First seen Nov 18, 2025 · Last updated Jun 16, 2026 · Updated 29 times
Summary
This study tests a new gene therapy for X-linked severe combined immunodeficiency (X-SCID), a rare immune disorder. Doctors take a person's own stem cells, fix a faulty gene using a technique called base editing, and return the cells to the body. The goal is to rebuild the immune system. The trial includes 18 people aged 3 and older and will follow them for 15 years.
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This is a summary of
the original study
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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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Locations
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National Institutes of Health Clinical Center
Bethesda, Maryland, 20892, United States
Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.