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In vivo somatic cell base editing and prime editing
Molecular Therapy ( IF 12.1 ) Pub Date : 2021-09-10 , DOI: 10.1016/j.ymthe.2021.09.002
Gregory A Newby 1 , David R Liu 1
Affiliation  

Recent advances in genome editing technologies have magnified the prospect of single-dose cures for many genetic diseases. For most genetic disorders, precise DNA correction is anticipated to best treat patients. To install desired DNA changes with high precision, our laboratory developed base editors (BEs), which can correct the four most common single-base substitutions, and prime editors, which can install any substitution, insertion, and/or deletion over a stretch of dozens of base pairs. Compared to nuclease-dependent editing approaches that involve double-strand DNA breaks (DSBs) and often result in a large percentage of uncontrolled editing outcomes, such as mixtures of insertions and deletions (indels), larger deletions, and chromosomal rearrangements, base editors and prime editors often offer greater efficiency with fewer byproducts in slowly dividing or non-dividing cells, such as those that make up most of the cells in adult animals. Both viral and non-viral delivery methods have now been used to deliver base editors and prime editors in animal models, establishing that base editors and prime editors can serve as effective agents for therapeutic genome editing in animals. This review summarizes examples of somatic cell (post-natal) base editing and prime editing and prospects for future development.

中文翻译:


体内体细胞碱基编辑和引物编辑



基因组编辑技术的最新进展放大了单剂量治愈许多遗传疾病的前景。对于大多数遗传性疾病,精确的 DNA 校正有望为患者提供最佳治疗。为了高精度地安装所需的 DNA 变化,我们的实验室开发了碱基编辑器 (BE),它可以纠正四种最常见的单碱基替换;以及 Prime 编辑器,可以在一段时间内安装任何替换、插入和/或删除。数十个碱基对。与涉及双链 DNA 断裂 (DSB) 并经常导致大量不受控制的编辑结果(例如插入和删除 (indels) 混合、较大删除和染色体重排)的核酸酶依赖性编辑方法相比,碱基编辑器和在缓慢分裂或非分裂细胞(例如构成成年动物大部分细胞的细胞)中,prime 编辑器通常可以提供更高的效率和更少的副产物。病毒和非病毒递送方法现已用于在动物模型中递送碱基编辑器和初等编辑器,证实碱基编辑器和初等编辑器可以作为动物治疗性基因组编辑的有效试剂。本综述总结了体细胞(产后)碱基编辑和prime编辑的实例以及未来发展的前景。
更新日期:2021-09-10
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