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GENOME EDITING

Production of foetal globin in adult monkeys

Nature Biomedical Engineering volume 3, pages 857–859 (2019)Cite this article

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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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Fig. 1: Gene editing of haematopoietic stem cells for inducing the production of foetal haemoglobin in rhesus macaques.

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Authors and Affiliations

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

    Daniel E. Bauer

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

    Daniel E. Bauer

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

    Daniel E. Bauer

  4. Broad Institute, MIT and Harvard, Cambridge, MA, USA

    Daniel E. Bauer

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

    Daniel E. Bauer

Authors
  1. Daniel E. Bauer
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Correspondence to Daniel E. Bauer.

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Competing interests

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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