![\"\"](\"https:\/\/www.newsbeep.com\/ie\/wp-content\/uploads\/2025\/09\/Illustration-of-spherical-nucleic-acid-SNA-nanoparticle-carrying-CRISPR-cargo-Image-by-the-Mirkin-Re.jpeg\")
Illustration of spherical nucleic acid SNA nanoparticle carrying CRISPR cargo \u2013 Image by the Mirkin Research Group<\/p>\n Illustration of spherical nucleic acid SNA nanoparticle carrying CRISPR cargo \u2013 Image by the Mirkin Research Group<\/p>\n
University scientists in Chicago have developed a new nanostructure that triples CRISPR\u2019s ability to enter cells, potentially unlocking even more power to treat genetic diseases.<\/p>\n
With the power to rewrite the genetic code that underlies countless diseases, CRISPR holds immense promise to revolutionize medicine.<\/p>\n
Now, Northwestern University chemists have unveiled a new type of nanostructure that dramatically improves CRISPR delivery and potentially extends its scope of utility.<\/p>\n
CRISPR machinery cannot enter cells by itself. It always needs a delivery vehicle. Called \u2018lipid nanoparticle spherical nucleic acids\u2019 (LNP-SNAs), these tiny structures carry the full set of CRISPR editing tools\u2014like Cas9 enzymes.<\/p>\n
They\u2019re wrapped in a dense, protective shell of DNA that dictates which organs and tissues the nanoparticles travel to and makes it easier for them to enter cells.<\/p>\n
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.<\/p>\n
The new nanostructures also improved the success rate of precise DNA repairs by more than 60% compared to current methods.<\/p>\n
Their study, published in the<\/a> Proceedings of the National Academy of Sciences, paves the way for safer, more reliable genetic medicines and underscores the importance of how a nanomaterial\u2019s structure\u2014rather than its ingredients alone\u2014can determine its potency.<\/p>\n This principle underlies structural nanomedicine, an emerging field pioneered by Northwestern\u2019s Chad A. Mirkin and his colleagues and pursued by hundreds of researchers around the world.<\/p>\n \u201cCRISPR is an incredibly powerful tool that could correct defects in genes to decrease susceptibility to disease and even eliminate disease itself,\u201d said Mirkin, who led the new study.<\/p>\n \u201cBut it\u2019s difficult to get CRISPR into the cells and tissues that matter. Reaching and entering the right cells\u2014and the right places within those cells\u2014requires a minor miracle.<\/p>\n \u201cWe aimed to maximize CRISPR\u2019s efficiency and expand the number of cell and tissue types that we can deliver it to.\u201d<\/p>\n MORE GOOD CRISPR NEWS: Scientists sometimes use viral vectors as the as a delivery vehicle. Naturally good at sneaking into cells, viruses are efficient, but they can cause the human body to mount an immune response, leading to painful or even dangerous side effects.<\/p>\n LNPs, on the other hand, are safer but inefficient. They tend to get stuck in endosomes, or compartments within the cell, where they cannot release their cargo.<\/p>\n \u201cOnly 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,\u201d Mirkin said in a media release<\/a>.<\/p>\n To overcome this barrier, Mirkin\u2019s team turned to SNAs, which are globular \u2014 rather than linear \u2014 forms of DNA and RNA previously invented in Mirkin\u2019s lab at Northwestern.<\/p>\n The spherical genetic material surrounds a nanoparticle core, which can be packed with cargo. Roughly 50 nanometers in diameter, the tiny structures possess a proven ability to enter cells for targeted delivery. Seven SNA-based therapies are already in human clinical trials, including a Phase 1b\/2 clinical trial for solid tumors being developed by Flashpoint Therapeutics, a clinical-stage biotechnology startup.<\/p>\n In the new study, Mirkin\u2019s team started with an LNP core carrying the CRISPR machinery inside. Then, they decorated the particle\u2019s surface with a dense layer of short strands of DNA. Because the DNA can interact with a cell\u2019s surface receptors, cells easily absorb SNAs. The DNA also can be engineered with sequences that target specific cell types, making delivery more selective.<\/p>\n
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