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New Research Promises Potential Treatment for Hereditary Blindness

Using the same mechanism as the COVID-19 vaccine, researchers have found a way to spark activity in mutated photoreceptor cells

Holden Galusha

Holden Galusha is the associate editor for Lab Manager. He was a freelance contributing writer for Lab Manager before being invited to join the team full-time. Previously, he was the...

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Researchers from the Oregon State University College of Pharmacy have successfully demonstrated a potential treatment for hereditary blindness with the use of lipid nanoparticles (LNPs) and messenger RNA, the same technique upon which mRNA COVID-19 vaccines are built.

In a study published in Science Advancesthe authors describe the development of nanoparticles that can penetrate the neural retina and deliver mRNA directly to the photoreceptor cells, which instructs the cells to manufacture proteins needed for vision. With this new study, the researchers successfully sidestepped the main limitation of other LNP-based vision therapy approaches: successfully delivering the LNPs to the retina, located at the back of the eye.

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The mechanics of this solution are similar to how Coronavirus vaccines work; in the case of vaccines, mRNA carried through the body by LNPs instruct cells to replicate a harmless portion of the virus’ spike protein, initiating an immune response. In this new therapy for vision loss, LNPs equipped with peptides that “act as zip codes to deliver nanoparticles to the intended address” carry mRNA to the back of the retina, where the mRNA instructs mutated photoreceptor cells to manufacture the proteins needed for sight.

It should be noted that this technique only applies to individuals with inherited retinal degeneration, a class of vision impairments caused by hereditary mutations. These mutations prevent the photoreceptor cells from naturally creating the proteins needed for vision, which this therapy addresses.

While this technique has yet to be tested in humans, the scientists showed that LNPs armed with peptides successfully passed through barriers in the eye to reach the neural retina in both mice and non-human primates. With a $3.2 million grant from the National Eye Institute, the team hopes to continue their research. They intend to conduct research into using LNPs to deliver a gene editing tool that could replace faulty genes in photoreceptor cells with functioning genes.