CRISPR-Cas9 in Lipid Nanoparticles Found to Safely Treat Ovarian Cancer in Mice

CRISPR-Cas9 in Lipid Nanoparticles Found to Safely Treat Ovarian Cancer in Mice
For the first time, researchers safely and effectively treated cells and mice with ovarian cancer using the CRISPR-Cas9 gene-editing tool plus a newly developed lipid nanoparticle (LNP) delivery system, which targeted and destroyed the cancer cells by genetic manipulation.  This new therapeutic strategy has the potential to treat not only other types of cancer, but also genetic diseases and chronic viral infections, the researchers said. The findings were described in the study, “CRISPR-Cas9 genome editing using targeted lipid nanoparticles for cancer therapy,” published in the journal Science Advances. CRISPR-Cas9 is a molecular tool that can be used to precisely edit DNA in cells and has the potential to become a cancer treatment by permanently disrupting genes associated with tumor survival. This strategy would overcome some of the limitations of traditional therapies and improve treatment efficacy with fewer treatments. However, the current systems that deliver CRISPR-Cas9 result in low gene-editing efficiencies. Lipid nanoparticles (LNPs) are tiny fatty spheres that are clinically approved to deliver molecular therapies to diseased cells. Current LNPs are optimized for small molecules, but not for those needed to deliver CRISPR-Cas9 components, which are larger. Furthermore, while several delivery vehicles for CRISPR that target the liver have been reported, formulations designed for other tissues were less efficient.  “The CRISPR genome editing technology, capable of identifying and altering any genetic segment, has revolutionized our ability to disrupt, repair, or even replace genes in a personalized manner,” Dan Peer, PhD, the study's senior author, said in a press release. “Despite its extensive use in research, clinical im
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