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Mouse study supports feasibility of ocular gene replacement therapy
CLEVELAND — Concentrated DNA nanoparticles injected into the eyes of mice were effectively expressed by the retinal rod and cone cells, an experimental study found.
The results suggest that "genetic replacement therapy is feasible for various eye diseases," said Robert C. Moen, MD, PhD, in a press release announcing the study results.
Dr. Moen is president and chief executive officer of Copernicus Therapeutics, which is developing DNA nanoparticle technology for treating retinal disorders such as diabetic retinopathy, macular degeneration and other diseases that injure ganglion cells and the optic nerve.
Muna I. Naash, PhD, of University of Oklahoma Health Sciences Center, and colleagues investigated the ability of nanoparticles to non-virally transfer genes to ocular tissues. After injecting the DNA nanoparticles, the researchers found that 95% of retinal cells expressed the new DNA without any evidence of toxicity. In addition, the levels of expression produced were "almost equal to that of [rhodopsin], the highest expressed gene in the retina," the study authors said.
The study is published in the December 20 issue of Public Library of Science One.