Lipid Nanoparticles for Delivery of CRISPR Gene Editing Components.

IF 10.7 2区 材料科学 Q1 CHEMISTRY, PHYSICAL
Fan Wu, Nei Li, Yudian Xiao, Rohan Palanki, Hannah Yamagata, Michael J Mitchell, Xuexiang Han
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引用次数: 0

Abstract

Gene editing has emerged as a promising therapeutic option for treating genetic diseases. However, a central challenge in the field is the safe and efficient delivery of these large editing tools, especially in vivo. Lipid nanoparticles (LNPs) are attractive nonviral vectors due to their low immunogenicity and high delivery efficiency. To maximize editing efficiency, LNPs should efficiently protect gene editing components against multiple biological barriers and release them into the cytoplasm of target cells. In this review, the widely used CRISPR gene editing systems are first overviewed. Then, each component of LNPs, as well as their effects on delivery, are systematically discussed. Following this, the current LNP engineering strategies to achieve non-liver targeting are summarized. Finally, preclinical and clinical applications of LNPs for in vivo genome editing are highlighted, and perspectives for the future development of LNPs are provided.

用于递送CRISPR基因编辑组件的脂质纳米颗粒。
基因编辑已经成为治疗遗传性疾病的一种有前途的治疗选择。然而,该领域的一个核心挑战是安全有效地递送这些大型编辑工具,特别是在体内。脂质纳米颗粒(LNPs)由于其低免疫原性和高传递效率而成为极具吸引力的非病毒载体。为了最大限度地提高编辑效率,LNPs应该有效地保护基因编辑成分免受多种生物屏障的侵害,并将其释放到靶细胞的细胞质中。本文首先综述了目前广泛应用的CRISPR基因编辑系统。然后,系统地讨论了LNPs的各个组成部分及其对输送的影响。在此基础上,总结了目前实现非肝脏靶向的LNP工程策略。最后,重点介绍了LNPs在体内基因组编辑中的临床前和临床应用,并对LNPs的未来发展提出了展望。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
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来源期刊
Small Methods
Small Methods Materials Science-General Materials Science
CiteScore
17.40
自引率
1.60%
发文量
347
期刊介绍: Small Methods is a multidisciplinary journal that publishes groundbreaking research on methods relevant to nano- and microscale research. It welcomes contributions from the fields of materials science, biomedical science, chemistry, and physics, showcasing the latest advancements in experimental techniques. With a notable 2022 Impact Factor of 12.4 (Journal Citation Reports, Clarivate Analytics, 2023), Small Methods is recognized for its significant impact on the scientific community. The online ISSN for Small Methods is 2366-9608.
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