基因组编辑
清脆的
基因
Cas9
突变
遗传学
计算生物学
双股
生物
RNA编辑
计算机科学
DNA修复
基因表达
作者
Zhihao Liu,Dong Guo,Dawei Wang,Jinglin Zhou,Qi Chen,Junzhong Lai
标识
DOI:10.1096/fj.202401692r
摘要
Abstract Genetic mutations significantly contribute to the onset of diseases, with over half of the cases caused by single‐nucleotide mutations. Advances in gene editing technologies have enabled precise editing and correction of mutated genes, offering effective treatment methods for genetic disorders. CRISPR/Cas9, despite its power, poses risks of inducing gene mutations due to DNA double‐strand breaks (DSB). The advent of base editing (BE) and prime editing (PE) has mitigated these risks by eliminating the hazards associated with DNA DSBs, allowing for more precise gene editing. This breakthrough lays a solid foundation for the clinical application of gene editing technologies. This review discusses the principles, development, and applications of PE gene editing technology in various genetic mutation‐induced diseases.
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