Vision Deficit in Retinitis Pigmentosa Mice Corrected With DNA Nanoparticles
Copernicus Therapeutics, Inc. announced today that a research team at University of Oklahoma Health Sciences Center, led by Dr. Muna Naash, Professor of Cell Biology, demonstrated that Copernicus’ DNA nanoparticles corrected vision defects in a mouse model of retinitis pigmentosa (RP). These findings were presented at the American Society of Gene Therapy meeting in Seattle, WA. Mutations in genes important in the biology of vision cause RP, a common genetic form of visual impairment affecting nearly 70,000 patients in the United States.
“These exciting results demonstrate that delivery of normal copies of genes into photoreceptor cells can correct vision defects in RP,” said Mark Cooper, M.D., Senior Vice President of Science and Medical Affairs. “In addition to the promise of providing corrective therapy for genetic diseases such as retinitis pigmentosa, nucleic acid nanoparticles may also offer the potential to provide effective treatments for more complex disorders such as diabetic retinopathy, macular degeneration, and various diseases that injure ganglion cells and the optic nerve.”
“We are pleased by the promise of these results and look forward to moving these studies forward to a potential human clinical trial,” said Dr. Naash.
“Gene therapy holds great potential for treating and possibly curing a variety of vision-robbing retinal degenerative diseases,” says Stephen Rose, Ph.D., Chief Research Officer, Foundation Fighting Blindness. “Dr. Naash and Copernicus are demonstrating that nanoparticles show excellent potential for safely and effectively delivering therapeutic genes to the retina.” Dr. Naash’s work was funded in part by the Foundation Fighting Blindness.
About Copernicus, the University of Oklahoma and the Foundation Fighting Blindness
Copernicus Therapeutics, Inc., a privately held biotechnology company, is dedicated to delivering the promise of nucleic acid therapeutics. The same technology that is being tested for its ability to deliver corrective nucleic acids for various blinding disorders is being developed for introducing the cystic fibrosis gene to the lungs of CF patients. The Copernicus multi-component delivery platform can be used to develop nucleic acid therapies for numerous human diseases. Additional information about Copernicus is available at www.cgsys.com.
The University of Oklahoma is a doctoral degree-granting research university serving the educational, cultural, economic and health care needs of the state, region and nation. The OU Health Sciences Center, which is located in Oklahoma City, is one of only four comprehensive academic health centers in the nation with seven professional colleges. Additional information about University of Oklahoma Health Sciences Center is available at www.ouhsc.edu.
The urgent mission of the Foundation Fighting Blindness is to drive the research that will provide preventions, treatments and cures for people affected by retinitis pigmentosa (RP), macular degeneration, Usher syndrome, and the entire spectrum of retinal degenerative diseases. Visit www.FightBlindness.org for more information on the Foundation’s efforts.