New Approach to Deliver Hereditary Blindness Therapeutics

A technique for delivering messenger RNA into neural cells of the eye is described in Science Advances this week, marking a key step toward treating blindness associated with inherited retinal degenerations (IRDs). IRDs are a complex group of genetic disorders that arise from mutations in the more than 300 different genes necessary for photoreceptor and retinal pigment epithelium functioning. While a number of drugs for IRDs are under development and one has been approved in the US, they rely on adeno-associated viruses (AAVs) for the delivery of their therapeutic payloads, which have a number of limitations including immunogenicity and limited packaging capacity. In this week's study, a team led by scientists from Oregon State University looked to lipid nanoparticles (LNPs) as delivery vehicles, developing ones that can carry mRNA and use peptides to localize the drug to the neural retina, which had previously been inaccessible to LNPs. They demonstrate their approach in rodents and non-human primates, suggesting LNP-mRNA delivery could have clinical applications for IRDs