Published in Research

Nanotechnology targets blindness via gene therapy

This is editorially independent content
2 min read

Researchers from the Oregon Health & Science University and Oregon State University have created a new gene therapy approach to deliver messenger ribonucleic acid (mRNA) inside the eye.

Give me some background first.

The FDA approved the first gene therapy to treat an inherited form of blindness in 2017. The therapy (voretigene neparvovec-rzyl) is now sold under the Luxturna brand name and uses a modified version of the adeno-associated virus (AAV) to deliver gene-revising molecules. (via)

While the majority of today’s gene therapies use AAV, limitations still exist: its small size, inability to physically contain gene-editing machinery for some more complex mutations, and its inability to deliver mRNA—only DNA.

Tell me about this new therapy.

This new approach uses lipid nanoparticles (LNPs) as an alternative to send mRNA into the eye. From there, the mRNA is designed to create proteins that alter gene mutations known to harm vision.

Why lipid nanoparticles?

LNPs are the most clinically advanced nonviral platform for mRNA that, unlike AAV, don’t have size constraints. While AAVs can only deliver DNA—leading to gene-editing molecules continuing to be created, as a result, unplanned gene edits—LNPs deliver mRNA and keep gene-editing machinery open for only a short time.

Talk about the study.

Investigators intravitreally injected a nanoparticle-based gene therapy model into the eyes of mice and nonhuman primates. They examined the eyes using confocal microscopy, among other imaging techniques.

See here for the full study.

What did they find?

The retinal tissue of the nonhuman primate subjects indicated that the LNP shell reached photoreceptors and delivered mRNA successfully into the retina, resulting in green fluorescent protein.

Significance?

The study is the first instance where LNPs have shown to target photoreceptors in a nonhuman primate.

Anything else?

Investigators noted that they will continue to work on follow-up research to determine the exact amount of green fluorescent protein present in animal retinal models.