Prolonged Expression of a Lysosomal Enzyme in Mouse Liver After Sleeping Beauty Transposon-Mediated Gene Delivery: Implications for Non-Viral Gene Therapy of Mucopolysaccharidosis

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Background—The Sleeping Beauty (SB) transposon system is a non-viral vector system that can integrate precise sequences into chromosomes. We evaluated the SB transposon system as a tool for gene therapy of mucopolysaccharidosis (MPS) types I and VII…