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Genome editing

Gene correction for sickle cell disease hits its prime

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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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Correspondence to Daniel E. Bauer.

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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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