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Nanoparticles are a rapidly emerging technology that is appearing in emerging treatments for retinal diseases, cancer, HIV, and a variety of other conditions. These microscopic particles — which are like tiny rocks as small as one fifty-thousandth of an inch — are uniquely able to penetrate cells and tissue for the delivery of therapeutic genes and molecules. An investigative team funded by the Foundation Fighting Blindness demonstrated that nanoparticles may be a safe and effective way to deliver healthy genes to replace the abnormal genes that cause vision-robbing retinal degenerative diseases such as retinitis pigmentosa (RP). Muna Naash, Ph.D., University of Oklahoma, in collaboration with Copernicus Therapeutics (Cleveland, Ohio), used nanoparticles to restore vision in mice with RP. The nanoparticles were produced by compressing DNA with the corrective gene and coating it in a waxy, slippery substance. These particles were then injected into the retinas of the mice to deliver the gene. The treated mice showed both functional and structural improvement in their retinas. “Our results provide proof-of-principle that nanoparticles may be used as a therapeutic intervention for retinal degenerative disorders,” says Naash. Naash, who has been involved in retinal disease research for the last 25 years, has been conducting Foundation-funded nanoparticle investigations for the last three years. She received her doctorate degree from Baylor College of Medicine, and her master and bachelor degrees from the University of Baghdad. Copernicus is developing nanoparticle technology for a variety of medical applications including: retinal diseases, cancer, vaccinations, and cystic fibrosis. Naash and her collaborators will continue to evaluate nanoparticles in small and large animal models for safety and efficacy before moving into human clinical trials. She hopes that human studies can begin in a few years. “Nanoparticles are a technology that we are excited about,” says Stephen Rose, Ph.D., Chief Research Officer, Foundation Fighting Blindness. “They are showing good potential for safe and targeted gene delivery for different forms of retinal degenerative diseases. In addition, nanoparticles are being used to deliver small neuroprotective molecules in the eye with sustained, long-term release." The Foundation also funds gene therapy research, which employs man-made, therapeutic viruses to deliver genes to the retina. “We are investing in both viral and nanoparticle gene delivery research projects, because both strategies may have benefits in getting healthy genes to the retina,” says Rose. |
