LNP Delivers CRISPR Directly to Mouse Liver, Dramatically Cuts Cholesterol Levels for Months March 2, 2021 Scientists at Tufts University and the Broad Institute of Harvard and MIT have developed a lipid nanoparticle (LNP) technology that can package and deliver CRISPR gene editing machinery specifically to the liver. Their studies in mice demonstrated use of the LNP technology to shuttle CRISPR Cas9 mRNA and guide RNA directly to the liver, to knock down a gene called Angptl3. The strategy resulted in up to 57% reductions in blood cholesterol levels with the effects lasting for several months following a single injection. “We envision that with this LNP platform in hand, we could now make CRISPR a practical and safe approach to treat a broad spectrum of liver diseases or disorders,” said Zachary Glass, a graduate student in the lab of Qiaobing Xu, PhD, associate professor of biomedical engineering at Tufts’ School of Engineering and corresponding author of the study. Min Qiu, PhD, postdoctoral researcher in Xu’s lab and Glass are co-first authors of the study, in which they concluded: “The system we established here offers a clinically viable approach for liver-specific delivery of CRISPR-Cas9–based genome editing tools.” The paper, titled, “Lipid nanoparticle-mediated codelivery of Cas9 mRNA and single-guide RNA achieves liver-specific in vivo genome editing of Angptl3,” was published in