Revolutionizing genetic disease treatment: Recent technological advances in base editing

IF 4.7 3区工程技术 Q2 ENGINEERING, BIOMEDICAL Current Opinion in Biomedical Engineering Pub Date : 2023-06-02 DOI:10.1016/j.cobme.2023.100472

Tyler C. Daniel , Hongzhi Zeng , Emmanuel C. Osikpa , Xue Gao

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引用次数: 2

Abstract

Treating human genetic diseases has posed a significant and longstanding challenge to the medical community with traditional approaches often limited by their targeting scope, precision, and safety. In recent years, the development of Base Editors (BEs) has revolutionized the genome editing field, offering a precise and effective therapeutic option for correcting single nucleotide mutations responsible for more than half of human genetic diseases without causing unintended DNA damage. Since their invention in 2016, BEs have been successfully used in both research and clinical settings to treat many human genetic diseases, including sickle cell disease and amyotrophic lateral sclerosis. This review summarizes the most impactful developments in BE technology between 2020 and 2023, particularly emphasizing the methodology used to enhance this revolutionary tool. By offering a comprehensive overview of the latest advances in BE technology, we hope to provide a framework to accelerate future innovations in this field.

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革命性的遗传疾病治疗:碱基编辑的最新技术进展

治疗人类遗传疾病对医学界构成了一个重大而长期的挑战，传统方法往往受到其靶向范围、精度和安全性的限制。近年来，碱基编辑器(BEs)的发展彻底改变了基因组编辑领域，为纠正导致一半以上人类遗传疾病的单核苷酸突变提供了一种精确而有效的治疗选择，而不会造成意外的DNA损伤。自2016年发明以来，BEs已成功用于研究和临床治疗多种人类遗传疾病，包括镰状细胞病和肌萎缩性侧索硬化症。本文总结了2020年至2023年间BE技术最具影响力的发展，特别强调了用于增强这一革命性工具的方法。通过对BE技术最新进展的全面概述，我们希望提供一个框架来加速该领域的未来创新。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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