Gene therapy aims to fix immune defect in rare disease

NCT ID NCT03645486

First seen Apr 25, 2026 · Last updated Apr 25, 2026

Summary

This trial tests a gene therapy for Chronic Granulomatous Disease (CGD), a rare immune disorder that causes severe infections. The therapy uses a modified virus to deliver a working copy of the faulty gene into the patient's bone marrow cells. The goal is to restore immune function and reduce infections. The study involves 10 participants and will follow them for up to 15 years to check safety and effectiveness.

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Contacts and locations

Study contacts

  • Contact

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

Locations

  • Shenzhen Geno-immune Medical Institute

    RECRUITING

    Shenzhen, Guangdong, China, China

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

Conditions

Explore the condition pages connected to this study.