Bi-functionalized aminoguanidine-PEGylated periodic mesoporous organosilica nanoparticles: a promising nanocarrier for delivery of Cas9-sgRNA ribonucleoproteine

Pardis Rahimi Salekdeh; Leila Ma’mani; Javad Tavakkoly-Bazzaz; Hossein Mousavi; Mohammad Hossein Modarressi; Ghasem Hosseini Salekdeh

doi:10.1186/s12951-021-00838-z

Bi-functionalized aminoguanidine-PEGylated periodic mesoporous organosilica nanoparticles: a promising nanocarrier for delivery of Cas9-sgRNA ribonucleoproteine

Pardis Rahimi Salekdeh, Leila Ma’mani^*, Javad Tavakkoly-Bazzaz, Hossein Mousavi, Mohammad Hossein Modarressi, Ghasem Hosseini Salekdeh^*

^*Corresponding author for this work

Research output: Contribution to journal › Article › peer-review

4 Citations (Scopus)

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Abstract

Background: 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@PEG₁₅₀₀-PMO) as a potent and biocompatible nanocarrier for RNP delivery.

Results: 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@PEG₁₅₀₀-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.

Conclusions: 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.

Original language	English
Article number	95
Pages (from-to)	1-16
Number of pages	16
Journal	Journal of Nanobiotechnology
Volume	19
Issue number	1
DOIs	https://doi.org/10.1186/s12951-021-00838-z
Publication status	Published - 31 Mar 2021

Bibliographical note

Copyright the Author(s) 2021. Version archived for private and non-commercial use with the permission of the author/s and according to publisher conditions. For further rights please contact the publisher.

Keywords

Aminoguanidine Functionalized
Bi-functionalized Periodic Mesoporous Organosilica Nanoparticles
Cas9-sgRNA Ribonucleoproteine
Delivery nano-system
Gene editing
PEGylated
RNP

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10.1186/s12951-021-00838-z

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title = "Bi-functionalized aminoguanidine-PEGylated periodic mesoporous organosilica nanoparticles: a promising nanocarrier for delivery of Cas9-sgRNA ribonucleoproteine",

abstract = "Background: 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@PEG1500-PMO) as a potent and biocompatible nanocarrier for RNP delivery. Results: 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@PEG1500-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.Conclusions: 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.",

keywords = "Aminoguanidine Functionalized, Bi-functionalized Periodic Mesoporous Organosilica Nanoparticles, Cas9-sgRNA Ribonucleoproteine, Delivery nano-system, Gene editing, PEGylated, RNP",

author = "Salekdeh, {Pardis Rahimi} and Leila Ma{\textquoteright}mani and Javad Tavakkoly-Bazzaz and Hossein Mousavi and Modarressi, {Mohammad Hossein} and Salekdeh, {Ghasem Hosseini}",

note = "Copyright the Author(s) 2021. Version archived for private and non-commercial use with the permission of the author/s and according to publisher conditions. For further rights please contact the publisher. ",

year = "2021",

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day = "31",

doi = "10.1186/s12951-021-00838-z",

language = "English",

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Bi-functionalized aminoguanidine-PEGylated periodic mesoporous organosilica nanoparticles : a promising nanocarrier for delivery of Cas9-sgRNA ribonucleoproteine. / Salekdeh, Pardis Rahimi; Ma’mani, Leila; Tavakkoly-Bazzaz, Javad et al.

In: Journal of Nanobiotechnology, Vol. 19, No. 1, 95, 31.03.2021, p. 1-16.

Research output: Contribution to journal › Article › peer-review

TY - JOUR

T1 - Bi-functionalized aminoguanidine-PEGylated periodic mesoporous organosilica nanoparticles

T2 - a promising nanocarrier for delivery of Cas9-sgRNA ribonucleoproteine

AU - Salekdeh, Pardis Rahimi

AU - Ma’mani, Leila

AU - Tavakkoly-Bazzaz, Javad

AU - Mousavi, Hossein

AU - Modarressi, Mohammad Hossein

AU - Salekdeh, Ghasem Hosseini

N1 - Copyright the Author(s) 2021. Version archived for private and non-commercial use with the permission of the author/s and according to publisher conditions. For further rights please contact the publisher.

PY - 2021/3/31

Y1 - 2021/3/31

N2 - Background: 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@PEG1500-PMO) as a potent and biocompatible nanocarrier for RNP delivery. Results: 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@PEG1500-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.Conclusions: 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.

AB - Background: 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@PEG1500-PMO) as a potent and biocompatible nanocarrier for RNP delivery. Results: 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@PEG1500-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.Conclusions: 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.

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KW - Bi-functionalized Periodic Mesoporous Organosilica Nanoparticles

KW - Cas9-sgRNA Ribonucleoproteine

KW - Delivery nano-system

KW - Gene editing

KW - PEGylated

KW - RNP

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DO - 10.1186/s12951-021-00838-z

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JO - Journal of Nanobiotechnology

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SN - 1477-3155

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M1 - 95

ER -

Bi-functionalized aminoguanidine-PEGylated periodic mesoporous organosilica nanoparticles: a promising nanocarrier for delivery of Cas9-sgRNA ribonucleoproteine

Abstract

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