News Column

New Findings Reported from B.T. Harmon and Co-Authors Describe Advances in Gene Therapy

June 5, 2014

By a News Reporter-Staff News Editor at Gene Therapy Weekly -- Current study results on Biotechnology have been published. According to news reporting out of Cleveland, Ohio, by NewsRx editors, research stated, "Viral vectors are a commonly used method for gene therapy because of their highly efficient transduction of cells. However, many vectors have a small genetic capacity, and their potential for immunogenicity can limit their usefulness."

Our news journalists obtained a quote from the research, "Moreover, for disorders of the central nervous system (CNS), the need for invasive surgical delivery of viruses to the brain also detracts from their clinical applicability. Here, we show that intranasal delivery of unimolecularly compacted DNA nanoparticles (DNA NPs), which consist of single molecules of plasmid DNA encoding enhanced green fluorescent protein (eGFP) compacted with 10 kDa polyethylene glycol (PEG)-substituted lysine 30-mers (CK30PEG10k), successfully transfect cells in the rat brain. Direct eGFP fluorescence microscopy, eGFP-immunohistochemistry (IHC) and eGFP-ELISA all demonstrated eGFP protein expression 2 days after intranasal delivery. eGFP-positive cells were found throughout the rostral-caudal axis of the brain, most often adjacent to capillary endothelial cells. This localization provides evidence for distribution of the nasally administered DNA NPs via perivascular flow."

According to the news editors, the research concluded: "These results are the first report that intranasal delivery of DNA NPs can bypass the blood-brain barrier and transfect and express the encoded protein in the rat brain, affording a non-invasive approach for gene therapy of CNS disorders."

For more information on this research see: Intranasal administration of plasmid DNA nanoparticles yields successful transfection and expression of a reporter protein in rat brain. Gene Therapy, 2014;21(5):514-521. Gene Therapy can be contacted at: Nature Publishing Group, Macmillan Building, 4 Crinan St, London N1 9XW, England. (Nature Publishing Group -; Gene Therapy -

Our news journalists report that additional information may be obtained by contacting B.T. Harmon, Copernicus Therapeut Inc, Cleveland, OH, United States. Additional authors for this research include A.E. Aly, L. Padegimas, O. Sesenoglu-Laird, M.J. Cooper and B.L. Waszczak (see also Biotechnology).

Keywords for this news article include: Ohio, Biotechnology, Cleveland, DNA Research, Gene Therapy, Nanoparticle, United States, Bioengineering, Nanotechnology, Emerging Technologies, North and Central America

Our reports deliver fact-based news of research and discoveries from around the world. Copyright 2014, NewsRx LLC

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Source: Gene Therapy Weekly

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