Gene Therapy for Sickle-Cell Anemia Looks Promising—but It's Riddled with Controversy

Gene therapy is fighting to enter mainstream medicine. With sickle cell disease, the fight is heating up. Roughly two years ago, the FDA made the historic decision to approve the first gene therapy in the US, finally realizing the therapeutic potential of hacking our biological base code after decades of cycles of hope and despair. Other approvals soon followed, including Luxturna to target inherited blindness and Zolgensma, a single injection that could save children with a degenerative disease from their muscles wasting away and dying before the age of two.

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