Improving Therapeutic Potential of Non-viral Minimized DNA Vectors

Authors

Lirio M Arévalo-Soliz
Cinnamon L Hardee
Jonathan M Fogg
Nathan R Corman
Cameron Noorbakhsh
Lynn Zechiedrich

Publication Date

11-1-2020

Journal

Cell and Gene Therapy Insights

DOI

10.18609/cgti.2020.163

PMID

33953961

PMCID

PMC8095377

PubMedCentral® Posted Date

5-4-2021

PubMedCentral® Full Text Version

Author MSS

Published Open-Access

yes

Abstract

The tragic deaths of three patients in a recent AAV-based X-linked myotubular myopathy clinical trial highlight once again the pressing need for safe and reliable gene delivery vectors. Non-viral minimized DNA vectors offer one possible way to meet this need. Recent pre-clinical results with minimized DNA vectors have yielded promising outcomes in cancer therapy, stem cell therapy, stem cell reprograming, and other uses. Broad clinical use of these vectors, however, remains to be realized. Further advances in vector design and production are ongoing. An intriguing and promising potential development results from manipulation of the specific shape of non-viral minimized DNA vectors. By improving cellular uptake and biodistribution specificity, this approach could impact gene therapy, DNA nanotechnology, and personalized medicine.