Tuning the same delivery method used in the two mRNA COVID-19 vaccines could work to reduce cysts from a rare lung disease called lymphangioleiomyomatosis.
Researchers at the Tufts University School of Engineering are building a reputation for precision targeting in drug delivery. Their tools: tiny lipid-based nanoparticles (LNPs) fine tuned to latch on to specific tissues, organs, even cell types within the body. Their latest creation: LNPs that carry genetic instructions directly into the lungs.
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 Qi