Oregon Health & Science University and Oregon State University researchers are developing a new approach to deliver gene therapy to address blindness-causing genetic mutations. Their new 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 courtesy of OHSU/Tetiana Korzun)
Using nanotechnology that enabled mRNA-based COVID-19 vaccines, a new approach to gene therapy may improve how physicians treat inherited forms of blindness.
According to a Oregon Health and Science University (OHSU) news release collaborative team of researchers with OHSU and Oregon State University (OSU) 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,1 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.
“Our peptide is like a zip code, and the lipid nanoparticles are similar to an envelope that sends gene therapy in the...
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