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Nanoparticles Show Promise for Delivering Vision-Restoring

Genes to the Retina

By Ben A. Shaberman

Nanoparticles are a rapidly emerging technology that is appearing in countless everyday applications - from electronics to cleaning chemicals to treatments for a variety of diseases including cancer and HIV. Interest in these microscopic particles - which can be as small as one fifty-thousandth of the width of a hair - comes from their strength and durability, flexibility, and ability to penetrate cells and tissue.

An investigative team funded by the Foundation Fighting Blindness demonstrated that nanoparticles may be a safe and effective way to correct genetic variations 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 and her collaborators will continue to evaluate nanoparticles in small animals before moving their research forward toward human clinical trials.
"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.



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