Hepatocytes represent the primary target tissue for hereditary genetic diseases like Transthyretin Amyloidosis. In our latest white paper, we explore how our high-fidelity CRISPR-Cas24 editor achieves 94% gene silencing efficiency inside hepatocytes while avoiding off-target double-stranded breaks in adjacent tissues.
Key findings demonstrate that utilizing specific ionizable lipids in our nanoparticles increases the target tissue accumulation by 3.5-fold compared to standard commercial LNP packages.