Scientists test One-Time gene fix for devastating blood diseases
NCT ID NCT06647979
Summary
This early-stage study is testing the safety of a new, one-time gene-editing treatment for people with severe sickle cell disease or beta-thalassemia. Doctors take a patient's own blood stem cells, edit a specific gene in the lab to boost healthy fetal hemoglobin, and then return the cells after chemotherapy. The goal is to reduce or eliminate disease symptoms and the need for blood transfusions. The study will follow 10-14 teens and adults for two years to see if the treatment is safe and how well it works.
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Genom att skicka in godkänner du våra Användarvillkor
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Genom att skicka in godkänner du våra Användarvillkor
Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Boston Children's Hospital
RECRUITINGBoston, Massachusetts, 02115, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Conditions
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