Breakthrough Nanoparticle Boosts CRISPR Delivery

Quick Smiles:

• New SNA nanoparticles triple CRISPR delivery into cells, paving the way for better genetic treatments.
• These DNA-wrapped particles are safer and more efficient than traditional methods.
• Mirkin’s team is moving toward clinical trials to unlock CRISPR’s medical potential.

Researchers at Northwestern University have developed a nanoparticle that increases CRISPR’s ability to enter cells threefold, holding great promise for treating genetic conditions. This innovation, called lipid nanoparticle spherical nucleic acids (LNP-SNAs), makes it easier for CRISPR’s gene-editing tools to reach their cellular targets with less toxicity and increased efficiency.

The protective DNA shell on each nanoparticle helps direct the treatment to specific tissues, resulting in more effective and safer delivery compared to previous approaches.

Lab tests showed the new nanostructures deliver CRISPR up to three times more efficiently than standard systems, while also improving precise DNA repairs by over 60%.

“CRISPR is an incredibly powerful tool that could correct defects in genes to decrease susceptibility to disease and even eliminate disease itself,” said Chad A. Mirkin, who led the research.

Scientists have long faced challenges in delivering CRISPR safely and effectively, as viral vectors can cause immune reactions and traditional lipid particles often fail to reach their destination inside the cell. The new SNA architecture, however, enables rapid uptake into a wide variety of cell types and can be tailored to target specific ones.

“Only a fraction of the CRISPR machinery actually makes it into the cell and even a smaller fraction makes it all the way into the nucleus,” Mirkin explained. By surrounding nanoparticles with a DNA shell, his team maximized both entry and accuracy.

These advancements lay exciting groundwork for future therapies, with several SNA-based treatments already progressing through human clinical trials. Flashpoint Therapeutics, a university spin-out company, is working to bring this breakthrough technology to patients soon.

“By marrying two powerful biotechnologies — CRISPR and SNAs — we have created a strategy that could unlock CRISPR’s full therapeutic potential,” Mirkin said.

Stay tuned for more updates on innovations changing the future of medicine!

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