Investigators from the College of Pharmacy at Oregon State University have conclusively demonstrated a possible cure for hereditary blindness by using lipid nanoparticles (LNPs) and messenger RNA. This is the same approach that is used to construct mRNA COVID-19 vaccines.
The authors of a study that was recently published in Science Advances describe the development of nanoparticles that are capable of penetrating the neural retina and delivering messenger RNA (mRNA) directly to the photoreceptor cells. This instructs the photoreceptor cells to produce proteins that are necessary for vision. To properly transport the LNPs to the retina, which is situated in the rear of the eye, was the primary restriction of earlier LNP-based vision treatment techniques; however, the researchers with this new study were able to successfully circumvent this barrier.
The physics of this approach are similar to how Coronavirus vaccines operate; in the case of vaccinations, mRNA delivered across the body by LNPs direct cells to repeat a harmless component of the virus’ spike protein, eliciting an immune response. LNPs are used in this innovative treatment for visual loss. These LNPs are loaded with peptides that function as zip codes to transport nanoparticles to the desired destination. Once there, the mRNA directs mutant photoreceptor cells to produce the proteins that are required for sight.
It is important to highlight that this method can only be used on people who have inherited retinal degeneration, which is a category of visual abnormalities brought on by genetic defects passed down via families. Because of these mutations, the photoreceptor cells are unable to produce the proteins naturally that are necessary for vision; however, this treatment may correct this problem.
The researchers demonstrated that LNPs loaded with peptides were able to effectively cross past barriers in the eye of mice as well as non-human primates in order to reach the neuronal retina. However, this method has not yet been tried on humans. The National Eye Institute has awarded the group a grant of $3.2 million, which will hopefully allow them to continue their study. They propose to carry out research into the use of LNPs to provide a gene editing tool that has the potential to replace defective genes in photoreceptor cells with working ones.