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 Fiercebiotech. 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…