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专题聚焦第二部分:用于递送CRISPR/Cas9的固体脂质纳米粒的募集:抗癌基因编辑的初步评估

Special Focus Issue Part II: Recruitment of solid lipid nanoparticles for the delivery of CRISPR/Cas9: primary evaluation of anticancer gene editing.

作者信息

Akbaba Hasan, Erel-Akbaba Gulsah, Senturk Serif

机构信息

Department of Pharmaceutical Biotechnology, Faculty of Pharmacy, Ege University, Izmir, 35100, Turkey.

Department of Pharmaceutical Biotechnology, Faculty of Pharmacy, Izmir Katip Celebi University, Izmir, 35620, Turkey.

出版信息

Nanomedicine (Lond). 2021 May;16(12):963-978. doi: 10.2217/nnm-2020-0412. Epub 2021 May 10.

Abstract

The CRISPR/Cas9 system is a promising gene-editing tool for various anticancer therapies; however, development of a biocompatible, nonviral and efficient delivery of CRISPR/Cas9 expression systems remains a challenge. Solid lipid nanoparticles (SLNs) were produced based on pseudo and 3D ternary plots. Obtained SLNs and their complexes with PX458 plasmid DNA were characterized and evaluated in terms of cytotoxicity and transfection efficiency. SLNs were found to be nanosized, monodispersed, stable and nontoxic. Furthermore, they revealed similar transfection efficiency as the positive control. Overall, we have achieved a good SLN basis for CRISPR/Cas9 delivery and have the potential to produce SLNs with targeted anticancer properties by modifying production parameters and components to facilitate translating CRISPR/Cas9 into preclinical studies.

摘要

CRISPR/Cas9系统是一种用于各种抗癌治疗的很有前景的基因编辑工具;然而,开发一种生物相容性好、非病毒且高效的CRISPR/Cas9表达系统递送方法仍然是一项挑战。基于伪三元相图和三维三元相图制备了固体脂质纳米粒(SLNs)。对获得的SLNs及其与PX458质粒DNA的复合物进行了表征,并评估了其细胞毒性和转染效率。发现SLNs具有纳米尺寸、单分散、稳定且无毒的特点。此外,它们显示出与阳性对照相似的转染效率。总体而言,我们已经为CRISPR/Cas9递送建立了良好的SLN基础,并且有潜力通过修改生产参数和成分来生产具有靶向抗癌特性的SLNs,以促进将CRISPR/Cas9转化为临床前研究。

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