Liver-directed neonatal gene therapy prevents cardiac, bone, ear, and eye disease in mucopolysaccharidosis I mice.
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S. Cai | A. Hennig | R. Herati | A. Kovács | K. Parker Ponder | Yuli Liu | Lingfei Xu | J. Mosinger Ogilvie | Bin Wang | Annabel S. Fu | Sarah Chung | David Lee