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Gene Therapy
CRISPR Therapy (CTX001) for Thalassemia
Recruiting2 awardsPhase 3
Nashville, Tennessee
This trial will test a new way to treat a rare disease called transfusion-dependent thalassemia (TDT). The treatment involves using a person's own stem cells that have been modified using CRISPR-Cas9. The goal is to see if this is a safe and effective treatment for TDT.
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