Therapeutic adenine base editing of human hematopoietic stem cells

Jiaoyang Liao; Shuanghong Chen; Shenlin Hsiao; Yanhong Jiang; Yang Yang; Yuanjin Zhang; Xin Wang; Yongrong Lai; Daniel E. Bauer; Yuxuan Wu

doi:10.1038/s41467-022-35508-7

Nature Communications (Jan 2023)

Therapeutic adenine base editing of human hematopoietic stem cells

Jiaoyang Liao,
Shuanghong Chen,
Shenlin Hsiao,
Yanhong Jiang,
Yang Yang,
Yuanjin Zhang,
Xin Wang,
Yongrong Lai,
Daniel E. Bauer,
Yuxuan Wu

Affiliations

Jiaoyang Liao: Shanghai Frontiers Science Center of Genome Editing and Cell Therapy, Shanghai Key Laboratory of Regulatory Biology, Institute of Biomedical Sciences and School of Life Sciences, East China Normal University
Shuanghong Chen: Shanghai Frontiers Science Center of Genome Editing and Cell Therapy, Shanghai Key Laboratory of Regulatory Biology, Institute of Biomedical Sciences and School of Life Sciences, East China Normal University
Shenlin Hsiao: Shanghai Frontiers Science Center of Genome Editing and Cell Therapy, Shanghai Key Laboratory of Regulatory Biology, Institute of Biomedical Sciences and School of Life Sciences, East China Normal University
Yanhong Jiang: Shanghai Frontiers Science Center of Genome Editing and Cell Therapy, Shanghai Key Laboratory of Regulatory Biology, Institute of Biomedical Sciences and School of Life Sciences, East China Normal University
Yang Yang: Department of Hematology, The First Affiliated Hospital of Guangxi Medical University
Yuanjin Zhang: Shanghai Key Laboratory of Regulatory Biology, Institute of Biomedical Sciences and School of Life Sciences, East China Normal University
Xin Wang: Shanghai Key Laboratory of Regulatory Biology, Institute of Biomedical Sciences and School of Life Sciences, East China Normal University
Yongrong Lai: Department of Hematology, The First Affiliated Hospital of Guangxi Medical University
Daniel E. Bauer: Cancer and Blood Disorders Center, Dana-Farber Cancer Institute and Boston Children’s Hospital, Harvard Medical School
Yuxuan Wu: Shanghai Frontiers Science Center of Genome Editing and Cell Therapy, Shanghai Key Laboratory of Regulatory Biology, Institute of Biomedical Sciences and School of Life Sciences, East China Normal University

DOI: https://doi.org/10.1038/s41467-022-35508-7
Journal volume & issue: Vol. 14, no. 1
pp. 1 – 11

Abstract

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Here, Liao and colleagues apply adenine base editor ABE8e and its PAM-less variant ABE8e-SpRY to β-thalassemia patient hematopoietic stem cells in the form of ribonucleoprotein complexes, resulting in efficient long-term editing and β-thalassemia alleviation.

Published in Nature Communications

ISSN: 2041-1723 (Online)
Publisher: Nature Portfolio
Country of publisher: United Kingdom
LCC subjects: Science
Website: https://www.nature.com/ncomms/

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