Lipid nanoparticle delivers phenylalanine ammonia lyase mRNA to the liver leading to catabolism and clearance of phenylalanine in a phenylketonuria mouse model

Por Ramon Diaz-Trelles, Sharon Lee, Kristen Kuakini, Jenny Park, Adrian Dukanovic, Jose A. Gonzalez, Thanhchau Dam, Jae Heon Kim, Jerel Boyd Vega, Marciano Sablad, Priya P. Karmali, Kiyoshi Tachikawa, Padmanabh Chivukula
Publicado em 2022-09-01