Large-RNA screen identifies LC-1 lipid for gene editing in mice

The researchers behind a Nature Biotechnology paper say they screened delivery lipids using a large base-editor mRNA instead of the smaller reporter RNAs typically used. The screen identified LC-1, which enabled gene editing in mouse liver, lung and brain via different administration routes. Cas9 editing reached up to four times that of benchmark lipid nanoparticles. In cell studies, LC-1 released roughly twice as much large mRNA into the cytosol as benchmark formulations.