Liver-directed neonatal gene therapy prevents cardiac, bone, ear, and eye disease in mucopolysaccharidosis I mice. - Liu,Y.; Xu,L.; Hennig,A.K.; Kovacs,A.; Fu,A.; Chung,S.; Lee,D.; Wang,B.; Herati,R.S.; Mosinger,O.J.; Cai,S.R.; Parker,P.K. (Mol Ther, 2005)