Recent advances in prime editing technologies and their promises for therapeutic applications

被引:4
|
作者
Zeng, Hongzhi [1 ]
Daniel, Tyler C. [1 ]
Lingineni, Ananya [2 ]
Chee, Kelly [3 ]
Talloo, Komal [2 ]
Gao, Xue [1 ,4 ,5 ]
机构
[1] Rice Univ, Dept Chem & Biomol Engn, Houston, TX 77005 USA
[2] Rice Univ, Dept Bioengn, Houston, TX 77005 USA
[3] Rice Univ, Dept BioSci, Houston, TX 77005 USA
[4] Univ Penn, Dept Chem & Biomol Engn, Philadelphia, PA 19104 USA
[5] Univ Penn, Ctr Precis Engn Hlth, Philadelphia, PA 19104 USA
基金
美国国家卫生研究院; 美国国家科学基金会;
关键词
D O I
10.1016/j.copbio.2024.103071
中图分类号
Q5 [生物化学];
学科分类号
071010 ; 081704 ;
摘要
Prime editing (PE) is a groundbreaking genome editing technology offering unparalleled precision in targeted genome modifications and has great potential for therapeutic applications. This review delves into the core principles of PE and emphasizes its advancements, applications, and prospects. We begin with a brief introduction to PE principles, followed by a detailed examination of recent improvements in efficiency, precision, and the scale of feasible edits. These improvements have been made to the PE systems through guide RNA engineering, protein engineering, DNA repair pathway screening, chromosomal or epigenomic modification, and in silico design and optimization tools. Furthermore, we highlight in vivo studies showcasing the therapeutic potential of PE to model and treat genetic diseases. Moreover, we discuss PE's versatile applications in saturation genome editing and its applicability to nonhuman organisms. In conclusion, we address the challenges and opportunities linked with PE, emphasizing its profound impact on biological research and therapeutics.
引用
收藏
页数:11
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