Bubble boy disease gene therapy shows promise in early trial
NCT ID NCT03217617
First seen Nov 01, 2025 · Last updated Apr 30, 2026 · Updated 22 times
Summary
This early-stage trial tests a gene therapy for X-linked severe combined immunodeficiency (SCID-X1), a rare genetic disorder that leaves babies without a working immune system. The treatment uses a modified virus to deliver a corrected gene directly into the bloodstream. The goal is to help the body produce its own immune cells and fight infections.
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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: •••••@•••••
Locations
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Guilin Hospital of Chinese Traditional and Western Medicine
RECRUITINGGuilin, Guangxi, China
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Shenzhen Geno-immune Medical Institute
RECRUITINGShenzhen, Guangdong, 518000, China
Contact Phone: •••-•••-•••• Email: •••••@•••••
Conditions
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