Gene therapy offers new hope for babies with rare 'Bubble Boy' disease
NCT ID NCT03538899
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tests a gene therapy for Artemis-deficient severe combined immunodeficiency (ART-SCID), a life-threatening immune disorder. It involves taking the patient's own blood stem cells, adding a working copy of the faulty gene, and infusing them back after mild chemotherapy. The goal is to rebuild a functioning immune system and improve survival. The trial enrolls 24 newly diagnosed infants and follows them for 15 years.
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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
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University of California, San Francisco (UCSF) Children's Hospital
RECRUITINGSan Francisco, California, 94143, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Gene therapy offers hope for 'Bubble Boy' disease, but leukemia risk remains
- Milder stem cell transplant shows promise for kids with immune disorders
- New stem cell filter could make transplants safer for vulnerable patients
- New stem cell approach could fix immune systems in kids
- Scientists hunt for genetic clues behind rare immune disorder