Protein engineering technologies for development of next-generation genome editors

IF 4.7 3区工程技术 Q2 ENGINEERING, BIOMEDICAL

Current Opinion in Biomedical Engineering Pub Date : 2023-11-01 DOI:10.1016/j.cobme.2023.100514

Jackson Winter , Shraddha Shirguppe , Pablo Perez-Pinera

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引用次数: 0

Abstract

Base editors and prime editors have emerged as promising tools for the modeling and treatment of genetic diseases due to their ability to introduce targeted modifications in the genomic DNA of living cells. Several engineering approaches have been applied to improve their performance, ranging from simple protein design approaches to complex directed evolution schemes that can probe a vast landscape of mutational variants with minimal user intervention. These extensive efforts have led to new generations of editors with enhanced properties such as increased editing activity, tailored editing windows, increased targetability, smaller construct size for viral delivery, and decreased off-target effects. In this manuscript we review protein engineering technologies that have been recently utilized to create an ever-evolving landscape of high-performance gene editing tools specifically designed for genetic targets of interest and that have redefined what is possible in the field of precision medicine.

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开发下一代基因组编辑器的蛋白质工程技术

碱基编辑器和引物编辑器由于能够在活细胞的基因组DNA中引入靶向修饰，已成为建模和治疗遗传疾病的有前途的工具。已经应用了几种工程方法来提高它们的性能，从简单的蛋白质设计方法到复杂的定向进化方案，这些方案可以在最小的用户干预下探测突变变异的广阔景观。这些广泛的努力导致新一代的编辑器具有增强的属性，如增加的编辑活动，定制的编辑窗口，增加的针对性，更小的病毒传递结构尺寸，减少脱靶效应。在这篇手稿中，我们回顾了蛋白质工程技术，这些技术最近被用来创建一个不断发展的高性能基因编辑工具，专门为感兴趣的基因靶标设计，并重新定义了精准医学领域的可能性。

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来源期刊

Current Opinion in Biomedical Engineering Medicine-Medicine (miscellaneous)

CiteScore

8.60

自引率

2.60%

发文量