One-Shot gene therapy aims to cure devastating blood disorder
NCT ID NCT07436767
Summary
This early-stage study is testing a one-time gene therapy injection called KL003 for people with severe sickle cell disease. The treatment involves collecting a patient's own blood stem cells, modifying them in a lab to produce healthy hemoglobin, and then returning them to the patient. The main goals are to see if the modified cells successfully engraft and produce healthy red blood cells, and to monitor for any safety issues.
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