Groundbreaking gene editing trial aims to fix immune system in 'Bubble Boy' disease
NCT ID NCT06851767
First seen Nov 18, 2025 · Last updated May 14, 2026 · Updated 18 times
Summary
This study tests a new gene therapy for X-linked severe combined immunodeficiency (X-SCID), a rare disorder that leaves people without a working immune system. Researchers take a person's own blood stem cells, fix the faulty gene using a precise editing technique called base editing, and return the corrected cells to the body. The goal is to restore immune function and reduce the need for lifelong treatments. The trial involves 18 participants aged 3 and older and will follow 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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Locations
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National Institutes of Health Clinical Center
Bethesda, Maryland, 20892, United States
Conditions
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