Scientists use lipid nanoparticles to precisely target gene
editing to the liver

The genome editing technology CRISPR has emerged as a
powerful new tool that can change the way we treat disease. The
challenge when altering the genetics of our cells, however, is how
to do it safely, effectively, and specifically targeted to the
gene, tissue and organ that needs treatment. Scientists at Tufts
University and the Broad Institute of Harvard and MIT have
developed unique nanoparticles comprised of lipids—fat
molecules—that can package and deliver gene editing machinery
specifically to the liver. In a study published today in the
Proceedings of the National Academy of Sciences, they have shown
that they can use the lipid nanoparticles (LNPs) to efficiently
deliver the CRISPR machinery into the liver of mice, resulting in
specific genome editing and the reduction of blood cholesterol
levels by as much as 57%—a reduction that can last for at least
several months with just one shot.