AccScience Publishing / GTM / Volume 2 / Issue 1 / DOI: 10.36922/gtm.232
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Adenine base editing as a promising therapy for cardiovascular diseases

Luzi Yang1,2† Zihao Tao1† Xiaoteng Ma3† Xuanhui Zhang1 Yuxuan Guo1,2,4,5* Fei Gao3*
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1 Peking University Health Science Center, School of Basic Medical Sciences, Beijing, 100191, China
2 Peking University Institute of Cardiovascular Sciences, Beijing, 100191, China
3 Beijing Anzhen Hospital, Department of Cardiology, Capital Medical University, Beijing, 100029, China
4 Ministry of Education Key Laboratory of Molecular Cardiovascular Science, Beijing, 100191, China
5 Beijing Key Laboratory of Cardiovascular Receptors Research, Beijing, 100191, China
Global Translational Medicine 2023, 2(1), 232
Submitted: 25 October 2022 | Accepted: 27 January 2023 | Published: 14 February 2023
© 2023 by the Author(s). Licensee AccScience Publishing, Singapore. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution 4.0 International License ( )

Cardiovascular diseases (CVDs) are the leading causes of human death worldwide. Genetic variants serve as the major risk factor for CVDs, with limited therapeutic interventions in clinical practice. The recent surge of genome editing technologies offers the hope to correct genetic variants and to cure genetic diseases. Among the diverse genome editing tools, adenine base editors (ABEs) exhibit high efficiency, high specificity, and low off-target effects, successfully entering a clinical trial and demonstrating the tremendous potential to transform modern cardiovascular therapy. In this review, we summarize the basic knowledge about ABE, showcase three hallmark studies using ABE to ameliorate or treat CVDs in experimental animals, and lastly discuss about the key technical concerns that should be addressed to achieve the full potential of ABEs in the future.

Adenine base editor
Cardiovascular disease
Gene therapy
National Key R&D Program of China
National Natural Science Foundation of China
Beijing Nova Program
Postdoctoral Science Foundation of China

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Conflict of interest
No potential conflicts of interest were disclosed.
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Global Translational Medicine, Electronic ISSN: 2811-0021 Print ISSN: TBA, Published by AccScience Publishing