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The potential first gene therapy in the United States is being reviewed by a U.S. Food and Drug Administration expert panel.

Potential First U.S. Gene Therapy Now Under FDA Review

Tisagenlecleucel could be a one-time treatment for children and young adults with advanced leukemia
Gene therapy may benefit children and teens with Wiskott-Aldrich syndrome

Gene Tx May Benefit Children, Teens With Wiskott-Aldrich Sx

Patient's own blood stem cells are removed to correct the WAS gene, then injected back into the patient