Scientists test 'Base Editing' to cure 'Bubble Boy' disease
NCT ID NCT06851767
Summary
This early-phase trial is testing a new gene therapy for X-linked Severe Combined Immunodeficiency (X-SCID), a rare and life-threatening genetic disorder that severely weakens the immune system. Researchers will collect a participant's own blood stem cells, use a precise gene-editing technique called 'base editing' to repair the faulty gene causing the disease, and then return the corrected cells to the patient. The main goals are to see if this treatment is safe and if it can help rebuild a functional immune system in people aged 3 and older with X-SCID.
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Contacts and locations
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Locations
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National Institutes of Health Clinical Center
Bethesda, Maryland, 20892, United States
Conditions
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