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.
Small MethodsMaterials 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.