One-Time gene therapy aims to free patients from lifelong blood transfusions
NCT ID NCT06364774
Summary
This early-stage trial is testing a one-time gene therapy for people with severe beta-thalassemia, a blood disorder that requires lifelong regular blood transfusions. Doctors will collect a patient's own blood stem cells, add a healthy gene in the lab, and then return them after chemotherapy. The goal is to see if this treatment can safely reduce or eliminate the need for future transfusions.
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Genom att skicka in godkänner du våra Användarvillkor
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
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Contact
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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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