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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Format: | Article |
Language: | English |
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Elsevier
2023-06-01
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Series: | Molecular Therapy: Nucleic Acids |
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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. |
first_indexed | 2024-04-09T14:26:57Z |
format | Article |
id | doaj.art-328a004e2bb244c38b6637ccf2167d91 |
institution | Directory Open Access Journal |
issn | 2162-2531 |
language | English |
last_indexed | 2024-04-09T14:26:57Z |
publishDate | 2023-06-01 |
publisher | Elsevier |
record_format | Article |
series | Molecular Therapy: Nucleic Acids |
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 |