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Low-dose Gene Therapy Reduces the Frequency of Enzyme Replacement Therapy in a Mouse Model of Lysosomal Storage Disease

Alliegro M, Ferla R, Nusco E, De Leonibus C, Settembre C and Auricchio A

Low-dose Gene Therapy Reduces the Frequency of Enzyme Replacement Therapy in a Mouse Model of Lysosomal Storage Disease

Mol Ther 24: 2054-2063; advance online publication, October 25, 2016; doi:10.1038/mt.2016.181