Minicircle DNA-based gene therapy coupled with immune modulation permits long-term expression of α-L-iduronidase in mice with mucopolysaccharidosis type i

Mark J. Osborn, Ron T. McElmurry, Christopher J. Lees, Anthony P. Defeo, Zhi Ying Chen, Mark A. Kay, Luigi Naldini, Gordon Freeman, Jakub Tolar, Bruce R. Blazar

Research output: Contribution to journal › Article › peer-review

80 Scopus citations

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Biochemistry, Genetics and Molecular Biology