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碱基编辑:一种用于精确治疗遗传疾病的革命性技术。

Prime Editing: A Revolutionary Technology for Precise Treatment of Genetic Disorders.

作者信息

Li Mengyao, Lin Yi, Cheng Qiang, Wei Tuo

机构信息

State Key Laboratory of Stem Cell and Reproductive Biology, Institute of Zoology, Chinese Academy of Sciences, Beijing, China.

Key Laboratory of Organ Regeneration and Reconstruction, Institute of Zoology, Chinese Academy of Sciences, Beijing, China.

出版信息

Cell Prolif. 2025 Apr;58(4):e13808. doi: 10.1111/cpr.13808. Epub 2025 Feb 27.

Abstract

Genetic diseases have long posed significant challenges, with limited breakthroughs in treatment. Recent advances in gene editing technologies offer new possibilities in gene therapy for the treatment of inherited disorders. However, traditional gene editing methods have limitations that hinder their potential for clinical use, such as limited editing capabilities and the production of unintended byproducts. To overcome these limitations, prime editing (PE) has been developed as a powerful tool for precise and efficient genome modification. In this review, we provide an overview of the latest advancements in PE and its potential applications in the treatment of inherited disorders. Furthermore, we examine the current delivery vehicles employed for delivering PE systems in vitro and in vivo, and analyze their respective benefits and limitations. Ultimately, we discuss the challenges that need to be addressed to fully unlock the potential of PE for the remission or cure of genetic diseases.

摘要

长期以来,遗传疾病带来了重大挑战,治疗方面的突破有限。基因编辑技术的最新进展为遗传性疾病的基因治疗提供了新的可能性。然而,传统的基因编辑方法存在局限性,阻碍了它们在临床应用中的潜力,例如编辑能力有限以及产生意外的副产物。为了克服这些局限性,引导编辑(PE)已被开发成为一种用于精确高效基因组修饰的强大工具。在这篇综述中,我们概述了引导编辑的最新进展及其在遗传性疾病治疗中的潜在应用。此外,我们研究了目前在体外和体内递送引导编辑系统所使用的载体,并分析了它们各自的优点和局限性。最终,我们讨论了要充分释放引导编辑在缓解或治愈遗传疾病方面的潜力需要解决的挑战。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/d9e7/11969253/3a1e53664d31/CPR-58-e13808-g006.jpg

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