Intellia announces preclinical data showing effective CRISPR/Cas9 genome editing
Intellia Therapeutics announced that Cell Reports will publish at noon ET today its manuscript, "A single administration of CRISPR/Cas9 lipid nanoparticles achieves robust and persistent in vivo genome editing." The lipid nanoparticle delivery of Cas9 mRNA and sgRNA resulted in 97% reduction in mouse transthyretin protein levels in the liver, and the reduction was sustained for at least 12 months. The publication also documents that CRISPR/Cas9 components were undetectable in mice within three days after administration of Intellia's LNP delivery system. Researchers further demonstrated that Intellia's LNP technology is a similarly robust and effective delivery method for CRISPR/Cas9-mediated knockdown in rats, a higher rodent species.