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Innovators want pills to treat sickle cell disease. Can they match gene therapy?

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Although CRISPR-based gene therapy for sickle cell disease offers transformative outcomes, drugmakers are striving to develop treatments that are easy to manufacture and can reach much larger numbers of patients.

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  • 11 April 2024

    In the eighth paragraph, the sentence "They do so by disrupting an enhancer bound by the BCL11A erythroid-specific zinc-finger transcription factor, which suppresses γ-globin expression" has been removed from the HTML and PDF versions of the article as it was incorrect.

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Sheridan, C. Innovators want pills to treat sickle cell disease. Can they match gene therapy?. Nat Biotechnol 42, 347–350 (2024).

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