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Gene Therapy
Stem Cell Gene Therapy for Sickle Cell Disease
Recruiting1 awardPhase 1 & 2
Los Angeles, California
This trial tests a new treatment for adults with severe sickle cell disease using their own modified blood cells. The treatment aims to fix the genetic problem causing sickle cell disease and help patients produce healthy red blood cells. This approach could be better than current treatments and avoid complications from donor transplants. The new therapy involves modifying the patient's own blood cells to correct the genetic issue.
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