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Genome editing

Gene correction for sickle cell disease hits its prime

Nature Biomedical Engineering volume 7, pages 605–606 (2023)Cite this article

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Prime editing can efficiently rewrite the genetic mutation causing sickle cell disease, in haematopoietic stem cells from patients.

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Fig. 1: Prime editing at the β-globin gene locus corrects the HBBE6V mutation in human HSCs.

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Authors and Affiliations

  1. Division of Hematology/Oncology, Boston Children’s Hospital, Boston, MA, USA

    Sébastien Levesque & Daniel E. Bauer

  2. Department of Pediatric Oncology, Dana-Farber Cancer Institute, Boston, MA, USA

    Sébastien Levesque & Daniel E. Bauer

  3. Harvard Stem Cell Institute, Cambridge, MA, USA

    Sébastien Levesque & Daniel E. Bauer

  4. Broad Institute, Cambridge, MA, USA

    Sébastien Levesque & Daniel E. Bauer

  5. Department of Pediatrics, Harvard Medical School, Boston, MA, USA

    Sébastien Levesque & Daniel E. Bauer

Authors
  1. Sébastien Levesque
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  2. Daniel E. Bauer
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Correspondence to Daniel E. Bauer.

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The authors declare no competing interests.

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Levesque, S., Bauer, D.E. Gene correction for sickle cell disease hits its prime. Nat. Biomed. Eng 7, 605–606 (2023). https://doi.org/10.1038/s41551-023-01040-2

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