Health

Nanotechnology could improve gene therapy for blindness

Scientists have created lipid nanoparticles that can deliver mRNA to the retina, offering an opportunity for the development of additional gene therapies for the eye.

Studies have shown that lipid nanoparticles can reach photoreceptors in the eye and successfully deliver mRNA to the retina in mouse and primate studies. It was published of scientific progressResearchers at Oregon Health & Science University and Oregon State University have created an approach that uses lipid nanoparticles to deliver messenger ribonucleic acid or mRNA strands inside the eye.

This study opens the door to the development of additional gene therapies to treat eye diseases. Lipid nanoparticles can be used with mRNA to create proteins that edit genes that lead to blindness.

“More than 250 genetic mutations are associated with inherited retinal diseases, but only one has an approved gene therapy,” said a scientist at the OHSU Casey Eye Institute. . Press release“By improving the techniques used in gene therapy, we can offer more treatment options to prevent blindness. Our findings suggest that lipid nanoparticles can help do just that.” is showing.”

The only gene therapy approved to treat eye disease is Luxturna (voretigene neparvovec-rzyl), which was approved by the FDA in December 2017 to treat children and adults with hereditary blindness. Developed by Spark Therapeutics, Luxturna uses a modified version of adeno-associated virus (AAV) to deliver the biallelic RPE65 gene.

However, AAV vectors are small and can only deliver DNA. Lipid nanoparticles have no size limitation and can be used for mRNA delivery, thus avoiding off-target effects.

Another study author, Dr. Gaurav Sahay, an associate professor at the OSU College of Pharmacy and a future collaborator at the OHSU Casey Eye Institute, said the peptides they developed were able to precisely deliver mRNA to photoreceptors, cells, and so on. Until now, it has been impossible to target lipid nanoparticles.

In this study, Ryals, Sahay, and colleagues showed that a peptide-encrusted lipid nanoparticle shell could be targeted to photoreceptor cells in the retina. To demonstrate proof-of-concept, the researchers included mRNA along with instructions for making green fluorescent protein. When injected into the eyes of mice and non-human primates, the retinal tissue of the animals glowed green, demonstrating that the lipid nanoparticles reached the photoreceptors.

With a $3.1 million grant from the National Institutes of Health’s National Eye Institute, the researchers are working to develop therapeutics with mRNAs that carry the code for gene-editing molecules.

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Nanotechnology could improve gene therapy for blindness