One-Shot gene therapy aims to cure lifelong blood disorder
NCT ID NCT06364774
Summary
This early-stage study is testing a new, one-time gene therapy for people with severe beta-thalassemia, a blood disorder that requires lifelong blood transfusions. Doctors will collect a patient's own blood stem cells, add a healthy gene in the lab, and then return them. The goal is to see if this treatment is safe and can allow patients to live without needing regular transfusions.
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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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Children's Hospital of Philadelphia
RECRUITINGPhiladelphia, Pennsylvania, 19104, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Conditions
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