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Nanotechnology may improve gene therapy for blindness

New OHSU, OSU research uses lipid nanoparticles to target light-sensitive cells in the eye
January 11, 2023
3D illustration of nanoparticle inside an eye

New gene therapy method encases gene-editing molecules inside a shell of lipid nanoparticles. This scientific illustration shows the approach's lipid nanoparticle shells, which appear as fuzzy beige balls after they have been injected into the eye. Image credit: Tetiana Korzun, Oregon Health and Science University

Using nanotechnology that enabled mRNA-based COVID-19 vaccines, a new approach to gene therapy may improve how physicians treat inherited forms of blindness.

A collaborative team of researchers with Oregon Health & Science University and Oregon State University have developed an approach that uses lipid nanoparticles — tiny, lab-made balls of fat — to deliver strands of messenger ribonucleic acid, or mRNA, inside the eye. To treat blindness, the mRNA will be designed to create proteins that edit vision-harming gene mutations.

In a study published in Science Advances, the team demonstrates how its lipid nanoparticle delivery system targets light-sensitive cells in the eye, called photoreceptors, in both mice and nonhuman primates. The system’s nanoparticles are coated with a peptide that the researchers identified as being attracted to photoreceptors.

The scientists demonstrated that a peptide-covered lipid nanoparticle shell can be directed toward photoreceptor cells in the retina, tissue in the back of the eye that enables sight. As a first proof of concept, mRNA with instructions to make green fluorescent protein was placed inside nanoparticles.

After injecting this nanoparticle-based gene therapy model into the eyes of mice and nonhuman primates, the research team used a variety of imaging techniques to examine the treated eyes. The animals’ retinal tissue glowed green, illustrating that the lipid nanoparticle shell reached photoreceptors and that the mRNA it delivered successfully entered the retina and created green fluorescent protein. This research marks the first time that lipid nanoparticles are known to have targeted photoreceptors in a nonhuman primate.