Development of an mRNA replacement therapy for phenylketonuria - Perez-Garcia, Carlos G.; Diaz-Trelles, Ramon; Vega, Jerel Boyd; Bao, Yanjie; Sablad, Marciano; Limphong, Patty; Chikamatsu, Simon; Yu, Hailong; Taylor, Wendy; Karmali, Priya P.; Tachikawa, Kiyoshi; Chivukula, Padmanabh (Molecular therapy. Nucleic acids, 2022)
Keywords: hepatocytes; lipid nanoparticle; liver; LNP; mRNA; MT: Delivery Strategies; Original; PAH; phenylketonuria; PKU; replacement; therapy; lipid nanoparticle; liver; therapy; PAH; mRNA; PKU; MT: Delivery Strategies; phenylketonuria; replacement; hepatocytes; LNP