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). https://doi.org/10.1038/s41587-024-02179-2
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DOI: https://doi.org/10.1038/s41587-024-02179-2
