Improving cell and gene therapy safety and performance using next-generation Nanoplasmid vectors

The cell and gene therapy industry has employed the same plasmid technology for decades in vaccination, cell and gene therapy, and as a raw material in viral vector and RNA production. While canonical plasmids contain antibiotic resistance markers in bacterial backbones greater than 2,000 base pairs...

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Main Authors: James A. Williams, Patrick A. Paez
Format: Article
Language:English
Published: Elsevier 2023-06-01
Series:Molecular Therapy: Nucleic Acids
Subjects:
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253123000896
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author James A. Williams
Patrick A. Paez
author_facet James A. Williams
Patrick A. Paez
author_sort James A. Williams
collection DOAJ
description The cell and gene therapy industry has employed the same plasmid technology for decades in vaccination, cell and gene therapy, and as a raw material in viral vector and RNA production. While canonical plasmids contain antibiotic resistance markers in bacterial backbones greater than 2,000 base pairs, smaller backbones increase expression level and durability and reduce the cell-transfection-associated toxicity and transgene silencing that can occur with canonical plasmids. Therefore, the small backbone and antibiotic-free selection method of Nanoplasmid vectors have proven to be a transformative replacement in a wide variety of applications, offering a greater safety profile and efficiency than traditional plasmids. This review provides an overview of the Nanoplasmid technology and highlights its specific benefits for various applications with examples from recent publications.
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spelling doaj.art-328a004e2bb244c38b6637ccf2167d912023-05-04T04:12:21ZengElsevierMolecular Therapy: Nucleic Acids2162-25312023-06-0132494503Improving cell and gene therapy safety and performance using next-generation Nanoplasmid vectorsJames A. Williams0Patrick A. Paez1Research & Development, Aldevron, 4055 41st Avenue S, Fargo, ND 58104, USAResearch & Development, Aldevron, 4055 41st Avenue S, Fargo, ND 58104, USA; Corresponding author Patrick A. Paez, PhD, Research & Development, Aldevron, 4055 41st Avenue S, Fargo, ND 58104, USA.The cell and gene therapy industry has employed the same plasmid technology for decades in vaccination, cell and gene therapy, and as a raw material in viral vector and RNA production. While canonical plasmids contain antibiotic resistance markers in bacterial backbones greater than 2,000 base pairs, smaller backbones increase expression level and durability and reduce the cell-transfection-associated toxicity and transgene silencing that can occur with canonical plasmids. Therefore, the small backbone and antibiotic-free selection method of Nanoplasmid vectors have proven to be a transformative replacement in a wide variety of applications, offering a greater safety profile and efficiency than traditional plasmids. This review provides an overview of the Nanoplasmid technology and highlights its specific benefits for various applications with examples from recent publications.http://www.sciencedirect.com/science/article/pii/S2162253123000896MT: Delivery StrategiesplasmidminicircleNanoplasmidcell therapygene therapy
spellingShingle James A. Williams
Patrick A. Paez
Improving cell and gene therapy safety and performance using next-generation Nanoplasmid vectors
Molecular Therapy: Nucleic Acids
MT: Delivery Strategies
plasmid
minicircle
Nanoplasmid
cell therapy
gene therapy
title Improving cell and gene therapy safety and performance using next-generation Nanoplasmid vectors
title_full Improving cell and gene therapy safety and performance using next-generation Nanoplasmid vectors
title_fullStr Improving cell and gene therapy safety and performance using next-generation Nanoplasmid vectors
title_full_unstemmed Improving cell and gene therapy safety and performance using next-generation Nanoplasmid vectors
title_short Improving cell and gene therapy safety and performance using next-generation Nanoplasmid vectors
title_sort improving cell and gene therapy safety and performance using next generation nanoplasmid vectors
topic MT: Delivery Strategies
plasmid
minicircle
Nanoplasmid
cell therapy
gene therapy
url http://www.sciencedirect.com/science/article/pii/S2162253123000896
work_keys_str_mv AT jamesawilliams improvingcellandgenetherapysafetyandperformanceusingnextgenerationnanoplasmidvectors
AT patrickapaez improvingcellandgenetherapysafetyandperformanceusingnextgenerationnanoplasmidvectors