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One-shot gene fix aims to rebuild immune system in babies with bubble boy disease

NCT ID NCT03645460

Recruiting now Disease control Sponsor: Shenzhen Geno-Immune Medical Institute Source: ClinicalTrials.gov ↗

First seen Nov 01, 2025 · Last updated May 22, 2026 · Updated 32 times

Summary

This study tests a gene therapy given directly into the bloodstream for people with ADA-SCID, a severe genetic disorder that leaves them without a working immune system. The treatment uses a modified virus to deliver a working copy of the ADA gene, aiming to restore immune function. Ten participants will be monitored for safety and immune recovery over one year.

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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

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

Locations

  • Guilin Hospital of Chinese Traditional and Western Medicine

    RECRUITING

    Guilin, Guangxi, China

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • Shenzhen Geno-immune Medical Institute

    RECRUITING

    Shenzhen, Guangdong, 518000, China

    Contact Phone: •••-•••-•••• Email: •••••@•••••

Conditions

Explore the condition pages connected to this study.

ADENOSINE DEAMINASE SEVERE COMBINED IMMUNODEFICIENCY (ADA-SCID)