CRISPR ‘Prime Editing’ Breakthrough Cures Genetic Liver Disease in Mice
Breaking the Genetic “Spell” of a Liver Disease Phenylketonuria (PKU) is a hereditary metabolic liver disorder where a single DNA typo disrupts the PAH enzyme that breaks down phenylalanine, an amino acid nature.com fiercebiotech.com. Without a working PAH enzyme, phenylalanine builds up to toxic levels, causing brain damage, intellectual disability, and seizures if untreated. Newborn screening identifies PKU early, and patients are put on a strict lifetime diet limiting protein (to keep blood Phe 120–360 µM) pennmedicine.org fiercebiotech.com. Even with dietary control and medications, many patients struggle – foods high in protein are largely off-limits, and even newer drugs like