News Column

Study Findings from University of Oklahoma Provide New Insights into Gene Therapy

January 30, 2014

By a News Reporter-Staff News Editor at Gene Therapy Weekly -- Investigators publish new report on Biotechnology. According to news reporting originating in Oklahoma City, Oklahoma, by NewsRx journalists, research stated, "Given the number of monogenic ocular diseases and the number of non-monogenic degenerative ocular diseases for which gene therapy is considered as a treatment, the development of effective therapeutic delivery strategies for DNA is a critical research goal. In this work, nonviral nanoparticles (NPs) composed of glycol chitosan (GCS) and plasmid DNA (pDNA) were generated, characterized, and evaluated."

The news reporters obtained a quote from the research from the University of Oklahoma, "These particles are stable, do not aggregate in saline, are resistant to DNases, and have a hydrodynamic diameter of approximately 250nm. Furthermore, the plasmid in these NPs was shown to maintain its proper conformation and can be released and expressed inside the cell. To determine whether these NPs would be suitable for intraocular use, pDNA carrying the ubiquitously expressed CBA-eGFP expression cassette was compacted and subretinally injected into adult wild-type albino mice. At day14 post-injection (PI), substantial green fluorescent protein (GFP) expression was observed exclusively in the retinal pigment epithelium (RPE) in eyes treated with GCS NPs but not in those treated with uncompacted pDNA or vehicle (saline). No signs of gross retinal toxicity were observed, and at 30days PI, there was no difference in electroretinogram function between GCS NP-, pDNA-, or vehicle-treated eyes."

According to the news reporters, the research concluded: "These results suggest that with further development, GCS NPs could be a useful addition to the available repertoire of genetic therapies for the treatment of RPE-associated diseases."

For more information on this research see: Synthesis and Characterization of Glycol Chitosan DNA Nanoparticles for Retinal Gene Delivery. Chemmedchem, 2014;9(1):189-196. Chemmedchem can be contacted at: Wiley-V C H Verlag Gmbh, Boschstrasse 12, D-69469 Weinheim, Germany. (Wiley-Blackwell -; Chemmedchem -

Our news correspondents report that additional information may be obtained by contacting R.N. Mitra, University of Oklahoma, Hlth Sci Center, Dept. of Cell Biol, Oklahoma City, OK 73104, United States. Additional authors for this research include Z.C. Han, M. Merwin, M. Al Taai, S.M. Conley and M.I. Naash (see also Biotechnology).

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

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Source: Gene Therapy Weekly

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