Amelioration of the neurological phenotype in PKU mouse model emphasizes the potential of base editing for metabolic diseases

Gene editing has emerged as a promising therapeutic strategy for inborn errors of metabolism. New techniques enable the correction of disease-causing gene variants in living cells, or even in vivo in animal models or human individuals. Several metabolic diseases have already been targeted with lipid nanoparticle (LNP)-mediated base editing that efficiently enters the liver (Figure 1). However, the clinical phenotype often includes other organs; specifically, neurological symptoms are common, even if the defect in enzyme function is in the hepatocytes.