Doctors test One-Time gene fix for 'Bubble Boy' disease
NCT ID NCT03538899
Summary
This study is testing a new gene therapy for a rare and life-threatening immune disorder in babies called Artemis-deficient SCID. Doctors take a child's own blood stem cells, use a virus to insert a healthy copy of the faulty gene, and then return the corrected cells. The goal is to see if this one-time treatment can safely give the child a working immune system for life, especially when a matched sibling donor for a traditional transplant is not available.
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Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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University of California, San Francisco (UCSF) Children's Hospital
RECRUITINGSan Francisco, California, 94143, United States
Conditions
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