Gene therapy offers hope for boys born without immune systems
NCT ID NCT01410019
First seen Nov 01, 2025 · Last updated Apr 30, 2026 · Updated 24 times
Summary
This trial tested a gene therapy to treat X-linked severe combined immunodeficiency (SCID-X1), a rare genetic disorder that leaves boys without a working immune system. Five boys received a corrected copy of their faulty gene using a self-inactivating retroviral vector. The goal was to restore their immune defenses so they could fight infections normally, without needing 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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Locations
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Hopital Necker
Paris, 75015, France
Conditions
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