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

James A Williams; Patrick A Paez

doi:10.1016/j.omtn.2023.04.003

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

Mol Ther Nucleic Acids. 2023 Apr 7:32:494-503. doi: 10.1016/j.omtn.2023.04.003. eCollection 2023 Jun 13.

Authors

James A Williams¹, Patrick A Paez¹

Affiliation

¹ Research & Development, Aldevron, 4055 41st Avenue S, Fargo, ND 58104, USA.

Abstract

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.

Keywords: AAV; CRISPR-cas9; MT: Delivery Strategies; Nanoplasmid; adeno-associated virus; cell therapy; gene therapy; homology-directed repair; minicircle; plasmid.

Publication types

Review