LNP-SNAs are structures with a DNA shell that encapsulates CRISPR components such as Cas9 enzymes, guide RNAs, and DNA repair templates, enhancing targeting, cellular entry, and reducing toxicity.

The innovation exemplifies the synergy of nanotechnology and gene editing, offering broad potential applications across different tissues and cell types.

In vitro tests across multiple human and animal cell types confirmed higher gene modification rates, increased precise homologous recombination, and decreased toxicity, with sequencing validating these improvements.

Northwestern’s team plans to further validate the platform in disease models and aims to move toward clinical trials through biotech spin-out Flashpoint Therapeutics, with ongoing testing in animal models to confirm safety and efficacy.

Future efforts include extending in vivo testing and accelerating clinical translation, leveraging commercialization pathways to bring this technology closer to real-world therapeutic use.

Published in the Proceedings of the National Academy of Sciences, this research underscores the critical role of nanostructure design in enhancing nanomedicine efficacy.

This advancement addresses key challenges in CRISPR delivery, marking a major step toward safer, more effective gene therapies by improving targeted delivery and reducing immune responses.

Researchers at Northwestern University have developed a groundbreaking lipid nanoparticle spherical nucleic acid (LNP-SNA) platform that significantly improves the delivery and efficiency of CRISPR gene editing.

This hybrid delivery system has demonstrated a threefold increase in gene editing efficiency over traditional methods, with superior cellular internalization and lower toxicity in various cell types.

Compared to existing platforms, the LNP-SNA technology outperforms in multiple metrics and features a modular design adaptable for treating a wide range of diseases, including blood disorders and cancer.

Since SNAs are already in clinical trials for cancer and skin conditions, this technology holds promise for expanding the delivery of genetic therapies to broader medical applications.

The DNA shell not only protects CRISPR components but also allows for modular customization to target specific tissues or cell types, broadening therapeutic possibilities such as genetic disorder correction and neurological treatments.