New gene therapy offers hope for babies born without an immune system
NCT ID NCT03538899
Summary
This study is testing a new gene therapy for children with Artemis-SCID, a rare and life-threatening disorder where babies are born without a working immune system. Doctors take a child's own blood stem cells, use a modified virus to insert a healthy copy of the missing gene, and then return the corrected cells to the patient. The goal is to safely rebuild a lasting immune system without the need for a donor transplant, potentially offering a better treatment option for these children.
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Genom att skicka in godkänner du våra Användarvillkor
Contacts and locations
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Genom att skicka in godkänner du våra Användarvillkor
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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