Sickle-cell anemia gene therapy

Access through your institution Buy or subscribe _Nature_ 595, 295–302 (2021) The emergence of highly efficient gene-editing technologies has shown promise for developing new therapeutic

tools for human genetic disorders. Although past studies using CRISPR–Cas9 have yielded encouraging results, by-products such as chromosomal mutations from Cas9-mediated double-strand breaks

hinder clinical translation. Adenine base editors (ABEs) can efficiently convert A–T base pairs to G–T pairs when guided to a site of interest by catalytically impaired CRISPR–Cas9, and

were developed through directed evolution of a transfer-RNA adenosine deaminase. Newby et al. have now demonstrated durable editing of the sickle-cell disease allele in the β-globin gene

into a non-pathogenic variant in human-patient hematopoietic stem and progenitor cells that were transplanted into mice. Editing successfully mediated phenotypic rescue, thus suggesting a

potential one-time ABE treatment for sickle-cell disease. This is a preview of subscription content, access via your institution ACCESS OPTIONS Access through your institution Access Nature

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AND AFFILIATIONS * Nature Genetics https://www.nature.com/natgenet Ornob Alam Authors * Ornob Alam View author publications You can also search for this author inPubMed Google Scholar

CORRESPONDING AUTHOR Correspondence to Ornob Alam. RIGHTS AND PERMISSIONS Reprints and permissions ABOUT THIS ARTICLE CITE THIS ARTICLE Alam, O. Sickle-cell anemia gene therapy. _Nat Genet_

53, 1119 (2021). https://doi.org/10.1038/s41588-021-00918-8 Download citation * Published: 06 August 2021 * Issue Date: August 2021 * DOI: https://doi.org/10.1038/s41588-021-00918-8 SHARE

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