Gene therapy trial aims to fix 'Bubble Boy' disease in newborns
NCT ID NCT01512888
First seen Nov 10, 2025 · Last updated Jun 23, 2026 · Updated 35 times
Summary
This study tests a gene therapy for infants with X-linked severe combined immunodeficiency (SCID-X1), a life-threatening condition where babies lack a working immune system. Researchers take the baby's own bone marrow stem cells, add a normal copy of the faulty gene using a lentiviral vector, and infuse the cells back after a mild chemotherapy. The goal is to see if this approach is safe and can restore immune function, offering an alternative to stem cell transplants from a donor.
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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.
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
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Locations
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Seattle Children's Research Institute
Seattle, Washington, 98101, United States
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St. Jude Children's Research Hospital
Memphis, Tennessee, 38105, United States
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University of California-San Francisco
San Francisco, California, 94158, United States
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
lentiviral gene therapy (self-inactivating lentiviral vector with normal common gamma chain gene)
What this could lead to
If successful, this gene therapy could provide a new treatment option for infants with SCID-X1 who lack a suitable stem cell donor, potentially restoring their immune system without lifelong medication.
What could go wrong
This is an early-phase trial (Phase 1/2) with only 28 participants, so results may not apply broadly. There are risks from the chemotherapy (busulfan) used before gene transfer, and the therapy may fail to produce lasting immune recovery.
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.