Overview
- Northwestern researchers engineered lipid nanoparticle spherical nucleic acids that encapsulate Cas9, guide RNA, and a DNA repair template within a dense DNA shell.
- Across multiple human and animal cell types, the particles achieved up to threefold higher cellular uptake and editing efficiency with reduced toxicity versus standard lipid nanoparticles.
- The dense DNA coating both shields the CRISPR payload and promotes cellular entry, with sequence design enabling more selective tissue targeting.
- The work, published in Proceedings of the National Academy of Sciences, reported more than a 60% improvement in precise DNA repair in vitro.
- The team plans in vivo disease-model studies, and Northwestern spin-out Flashpoint Therapeutics is advancing the platform toward clinical development; no human data have been reported.