Liver-directed neonatal gene therapy prevents cardiac, bone, ear, and eye disease in mucopolysaccharidosis I mice

Yuli Liu, Lingfei Xu, Anne K. Hennig, Attila Kovacs, Annabel Fu, Sarah Chung, David Lee, Bin Wang, Ramin S. Herati, Judith Mosinger Ogilvie, Shi Rong Cai, Katherine Parker Ponder

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