"Called lipid nanoparticle spherical nucleic acids (LNP-SNAs), these tiny structures carry the full set of CRISPR editing tools - Cas9 enzymes, guide RNA and a DNA repair template - wrapped in a dense, protective shell of DNA. Not only does this DNA coating shield its cargo, but it also dictates which organs and tissues the LNP-SNAs travel to and makes it easier for them to enter cells."
"In lab tests across various human and animal cell types, the LNP-SNAs entered cells up to three times more effectively than the standard lipid particle delivery systems used for COVID-19 vaccines, caused far less toxicity and boosted gene-editing efficiency threefold. The new nanostructures also improved the success rate of precise DNA repairs by more than 60 percent compared to current methods."
"The study paves the way for safer, more reliable genetic medicines and underscores the importance of how a nanomaterial's structure - rather than its ingredients alone - can determine its potency. This principle underlies structural nanomedicine, an emerging field pioneered by Northwestern's Chad A. Mirkin, PhD, and his colleagues and pursued by hundreds of scientists around the world."
Lipid nanoparticle spherical nucleic acids (LNP-SNAs) encapsulate complete CRISPR machinery — Cas9 enzymes, guide RNA, and a DNA repair template — within a dense DNA shell that protects cargo, directs organ and tissue targeting, and facilitates cellular entry. Across multiple human and animal cell types, LNP-SNAs achieved up to threefold higher cellular uptake than standard lipid particles used in COVID-19 vaccines, produced substantially less toxicity, increased gene-editing efficiency threefold, and improved precise DNA-repair success by more than 60 percent. Structural design of nanomaterials governs delivery potency, informing the field of structural nanomedicine for genetic therapies.
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