A research team at Oregon State University confirmed the possibility of treating genetic retinal disease (IRD) by transmitting mRNA, which causes retinal disease, to photoreceptor cells with lipid nanoparticles, and published it in the journal Science Advances.
Genetic retinal disease is a rare intractable disease caused by mutations in genes responsible for the structure and function of retinal cells. Symptoms such as vision loss and night blindness appear, and in severe cases, blindness may occur. It is known to appear in the thousands.
The research team designed mRNA, which can make proteins that remove genetic mutations that induce vision damage, and put it in lipid nanoparticles. A peptide that moves toward the photoreceptor is attached to the surface of the lipid nanoparticles, which can carry mRNA to the photoreceptor.
Renee Lyres, an ophthalmology professor at Oregon State University, said, "More than 250 genetic mutations are known to be associated with genetic retinal disease, and there is only one approved gene therapy," adding, "Developing gene therapy can provide patients with more options to prevent blindness." He said, "Treatment using lipid nanoparticles will be one of them."
In 2017, the U.S. Food and Drug Administration (FDA) approved the first gene therapy drug "Luxterna" to treat blindness. This treatment uses an adenosine-related virus (AAV) as a carrier to deliver the treatment. However, only small DNA treatments can be delivered, and there is a possibility of errors, such as genetic correction in unintended areas.
On the other hand, lipid nanoparticles have no size constraints on the material to be delivered and can also deliver mRNA. The research team proved through mice and primates that lipid nanoparticles can reach retinal photoreceptor cells, a tissue located behind the eyes. A follow-up study is currently being conducted to quantify how many nanoparticles reach photoreceptors in animal retinal models.
Professor Lyres said, "We expect to be able to apply lipid nanoparticles to develop an improved delivery system than treatments using the adeno-related virus."
Writer: Yeyoung Jeon
(Picture from Unsplash)
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