Haematopoietic stem and progenitor cells, edited via CRISPR to mimic mutations ameliorating sickle-cell disease, engraft when transplanted in macaques and maintain foetal haemoglobin in circulation.
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D.E.B. is a co-inventor on various patents and patent applications related to therapeutic gene editing.
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Bauer, D.E. Production of foetal globin in adult monkeys. Nat Biomed Eng 3, 857–859 (2019). https://doi.org/10.1038/s41551-019-0475-3
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DOI: https://doi.org/10.1038/s41551-019-0475-3