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Acknowledgment to Reviewers of Bioengineering in 2020
 
 
Article

Stabilization of Poly (β-Amino Ester) Nanoparticles for the Efficient Intracellular Delivery of PiggyBac Transposon

Department of Biomedical Engineering and Chemical Engineering, University of Texas at San Antonio, One UTSA Circle, San Antonio, TX 78249, USA
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Bioengineering 2021, 8(2), 16; https://doi.org/10.3390/bioengineering8020016
Received: 30 December 2020 / Revised: 15 January 2021 / Accepted: 16 January 2021 / Published: 20 January 2021
The administration of gene-editing tools has been proposed as a promising therapeutic approach for correcting mutations that cause diseases. Gene-editing tools, composed of relatively large plasmid DNA constructs that often need to be co-delivered with a guiding protein, are unable to spontaneously penetrate mammalian cells. Although viral vectors facilitate DNA delivery, they are restricted by the size of the plasmid to carry. In this work, we describe a strategy for the stable encapsulation of the gene-editing tool piggyBac transposon into Poly (β-amino ester) nanoparticles (NPs). We propose a non-covalent and a covalent strategy for stabilization of the nanoformulation to slow down release kinetics and enhance intracellular delivery. We found that the formulation prepared by covalently crosslinking Poly (β-amino ester) NPs are capable to translocate into the cytoplasm and nuclei of human glioblastoma (U87MG) cells within 1 h of co-culturing, without the need of a targeting moiety. Once internalized, the nanoformulation dissociates, delivering the plasmid presumably as a response to the intracellular acidic pH. Transfection efficiency is confirmed by green fluorescence protein (GFP) expression in U87MG cells. Covalently stabilized Poly (β-amino ester) NPs are able to transfect ~55% of cells causing non-cytotoxic effects. The strategy described in this work may serve for the efficient non-viral delivery of other gene-editing tools. View Full-Text
Keywords: nanoparticles; plasmid delivery; gene therapies; non-viral vectors; crosslinking; layer-by-layer nanoparticles; plasmid delivery; gene therapies; non-viral vectors; crosslinking; layer-by-layer
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MDPI and ACS Style

Rodgers, T.; Muzzio, N.; Watson, C.; Romero, G. Stabilization of Poly (β-Amino Ester) Nanoparticles for the Efficient Intracellular Delivery of PiggyBac Transposon. Bioengineering 2021, 8, 16. https://doi.org/10.3390/bioengineering8020016

AMA Style

Rodgers T, Muzzio N, Watson C, Romero G. Stabilization of Poly (β-Amino Ester) Nanoparticles for the Efficient Intracellular Delivery of PiggyBac Transposon. Bioengineering. 2021; 8(2):16. https://doi.org/10.3390/bioengineering8020016

Chicago/Turabian Style

Rodgers, Tina, Nicolas Muzzio, Caleb Watson, and Gabriela Romero. 2021. "Stabilization of Poly (β-Amino Ester) Nanoparticles for the Efficient Intracellular Delivery of PiggyBac Transposon" Bioengineering 8, no. 2: 16. https://doi.org/10.3390/bioengineering8020016

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