One-Time gene fix could rescue 'Bubble Boy' disease

NCT ID NCT03645460

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tests a gene therapy given by IV for ADA-SCID, a rare and life-threatening immune disorder. The treatment uses a modified virus to deliver a working gene, aiming to restore the immune system. Ten participants will be monitored for safety and immune recovery over one year.

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Conditions

The condition(s) this trial relates to.

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Contacts and locations

Study contacts

  • Contact

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

Locations

  • Shenzhen Geno-immune Medical Institute

    RECRUITING

    Shenzhen, Guangdong, 518000, China

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