The Crispr components can’t naturally get into cells on their own, so Intellia uses a delivery system called lipid nanoparticles—essentially tiny fat bubbles—to ferry them to the liver. In Intellia’s trials, patients receive a one-time IV infusion of these Crispr-laden nanoparticles into the veins in their arms. Since blood passes through the liver, lipid nanoparticles can easily travel there from the bloodstream. In the liver, the nanoparticles are taken up by cells called hepatocytes. Once inside these cells, the nanoparticles break down and let Crispr get to work editing out the problematic gene.
In both diseases, a genetic mutation allows an aberrant protein to run amok and cause damage. In
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