Improving therapeutic potential of non-viral minimized DNA vectors

Published: 19 November 2020
Expert Insight
Lirio M Arévalo-Soliz,
Lirio M Arévalo-Soliz
Department of Molecular Virology and Microbiology, Baylor College of Medicine
Cinnamon L Hardee,
Cinnamon L Hardee
Department of Molecular Virology and Microbiology, Baylor College of Medicine
Jonathan M Fogg,
Jonathan M Fogg
Department of Molecular Virology and Microbiology, Baylor College of Medicine
Nathan R Corman,
Nathan R Corman
Rural Medical Education Program, University of Illinois College of Medicine
Cameron Noorbakhsh,
Cameron Noorbakhsh
Weiss School of Natural Sciences, Rice University
Lynn Zechiedrich
Lynn Zechiedrich
Department of Molecular Virology and Microbiology, Baylor College of Medicine
Lynn Zechiedrich is the Kyle and Josephine Morrow Chair and Professor in Molecular Virology and Microbiology at Baylor College of Medicine. She developed minivectors to study DNA, the enzymes that act on DNA, and the antibiotic and anticancer drugs that inhibit these enzymes. Minivectors also proved to be excellent gene therapy delivery vectors. Among other honors, she won a New Investigator Award from the Burroughs Wellcome Fund, a Curtis Hankamer Research Award, and funding from the Human Frontier Science Program. She is a Fellow of the National Academy of Inventors. She was Baylor College of Medicine’s BRASS Mentor of the Year in 2013. She holds two issued US patents and three issued foreign patents that are licensed to Twister Biotech, Inc., a company she founded in 2011, and has multiple patents pending. She has published more than 60 articles and book chapters and given over 170 invited talks. She served on numerous grant review committees, reviews for 40 different peer-reviewed journals, ranging from mathematics and physics to microbiology and gene therapy, and serves on multiple editorial boards.

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.

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