New Treatment for Hereditary Blindness Possible Using Nanoparticles and mRNA

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Scientists from the Oregon State University College of Pharmacy have demonstrated in animal models the possibility of using lipid nanoparticles and messenger RNA, the technology underpinning COVID-19 vaccines, to treat blindness associated with a rare genetic condition. Researchers developed nano

Nanoparticles entering neural retina. Credit: Image provided by Gaurav Sahay, OSU College of Pharmacy

Lipids are fatty acids and similar organic compounds including many natural oils and waxes. Nanoparticles are tiny pieces of material ranging in size from one- to 100-billionths of a meter. Messenger RNA delivers instructions to cells for making a particular protein. The scientists showed, in research involving mice and non-human primates, that LNPs equipped with peptides were able to pass through barriers in the eye and reach the neural retina – where light is turned into electric signals that the brain converts to images.

Sahay and Ryals have received a $3.2 million grant from the National Eye Institute to continue studying lipid nanoparticles’ promise in the treatment of hereditary blindness. They will lead research into using LNPs to deliver a gene editing tool that could delete bad genes in the photoreceptor cells and replace them with correctly functioning genes.

 

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