Liver-directed neonatal gene therapy prevents cardiac, bone, ear, and eye disease in mucopolysaccharidosis I mice
Titel:
Liver-directed neonatal gene therapy prevents cardiac, bone, ear, and eye disease in mucopolysaccharidosis I mice
Auteur:
Liu, Yuli Xu, Lingfei Hennig, Anne K. Kovacs, Attila Fu, Annabel Chung, Sarah Lee, David Wang, Bin Herati, Ramin S. Mosinger Ogilvie, Judith Cai, Shi-Rong Parker Ponder, Katherine