Department of Medical Genetics, School of Medicine, Tehran University of Medical Sciences, Tehran, Iran.
Department of Nanotechnology, Agricultural Biotechnology Research Institute of Iran (ABRII), Agricultural Research Education and Extension Organization (AREEO), Karaj, Iran.
J Nanobiotechnology. 2021 Mar 31;19(1):95. doi: 10.1186/s12951-021-00838-z.
There is a great interest in the efficient intracellular delivery of Cas9-sgRNA ribonucleoprotein complex (RNP) and its possible applications for in vivo CRISPR-based gene editing. In this study, a nanoporous mediated gene-editing approach has been successfully performed using a bi-functionalized aminoguanidine-PEGylated periodic mesoporous organosilica (PMO) nanoparticles (RNP@AGu@PEG-PMO) as a potent and biocompatible nanocarrier for RNP delivery.
The bi-functionalized MSN-based nanomaterials have been fully characterized using electron microscopy (TEM and SEM), nitrogen adsorption measurements, thermogravimetric analysis (TGA), X-ray powder diffraction (XRD), Attenuated Total Reflectance-Fourier Transform Infrared Spectroscopy (ATR-FTIR), and dynamic light scattering (DLS). The results confirm that AGu@PEG-PMO can be applied for gene-editing with an efficiency of about 40% as measured by GFP gene knockdown of HT1080-GFP cells with no notable change in the morphology of the cells.
Due to the high stability and biocompatibility, simple synthesis, and cost-effectiveness, the developed bi-functionalized PMO-based nano-network introduces a tailored nanocarrier that has remarkable potential as a promising trajectory for biomedical and RNP delivery applications.
人们对高效的 Cas9-sgRNA 核糖核蛋白复合物（RNP）的细胞内递呈及其在体内基于 CRISPR 的基因编辑中的潜在应用有着浓厚的兴趣。在这项研究中，一种纳米多孔介导的基因编辑方法已成功实现，使用双功能化的氨基胍-聚乙二醇化的周期性介孔有机硅（PMO）纳米颗粒（RNP@AGu@PEG-PMO）作为一种有效的、生物相容的 RNP 递送纳米载体。
使用电子显微镜（TEM 和 SEM）、氮气吸附测量、热重分析（TGA）、X 射线粉末衍射（XRD）、衰减全反射-傅里叶变换红外光谱（ATR-FTIR）和动态光散射（DLS）对基于 MSN 的双功能化纳米材料进行了全面表征。结果证实，AGu@PEG-PMO 可用于基因编辑，其效率约为 40%，通过 HT1080-GFP 细胞中 GFP 基因的敲低来衡量，而细胞形态没有明显变化。
由于其高稳定性和生物相容性、简单的合成和成本效益，所开发的基于双功能化 PMO 的纳米网络引入了一种定制化的纳米载体，作为生物医学和 RNP 递送应用的有前途的载体具有显著的潜力。
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