Gene therapy offers hope for babies with 'Bubble Boy' disease

NCT ID NCT05071222

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

Summary

This study tests a new gene therapy for children with a severe immune disorder called Artemis-SCID, where the body cannot fight infections. Doctors take the child's own blood stem cells, fix the faulty gene in a lab, and put the corrected cells back. The goal is to rebuild a working immune system without needing a bone marrow donor or lifelong medication.

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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

Locations

  • Department of Pediatric Immunology, Hematology and Rheumatology UIHR, Necker-Enfants Malades Hospital

    RECRUITING

    Paris, 75015, France