Scientists test One-Time gene fix for 'Bubble Boy' disease
NCT ID NCT05432310
Summary
This study is testing a one-time gene therapy for infants and children with a rare, life-threatening immune disorder called ADA-SCID. Doctors will collect the child's own blood stem cells, correct the faulty gene in a lab, and then return the cells after a mild chemotherapy. The main goal is to see if this treatment is safe and can allow the child's body to produce a needed enzyme and rebuild a working immune system without needing lifelong enzyme replacement shots.
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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, Los Angeles (UCLA)
RECRUITINGLos Angeles, California, 90095, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact Phone: •••-•••-•••• Email: •••••@•••••
Conditions
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