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Gene Therapy
CRISPR-Cas9 Gene Editing for Beta Thalassemia
Recruiting1 awardPhase 2 & 3
Manhattan, New York
This trial is testing a new treatment for people with a blood disorder called transfusion-dependent β-thalassemia. The treatment involves using CRISPR-Cas9 to modify the patient's own blood cells. The trial will evaluate the safety and efficacy of this treatment.
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