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Lentiviral-mediated gene therapy results in sustained expression of β-glucuronidase for up to 12 months in the gus(mps/mps) and up to 18 months in the gus(tm(L175F)Sly) mouse models of mucopolysaccharidosis type VII.
Derrick-Roberts AL, Pyragius CE, Kaidonis XM, Jackson MR, Anson DS, Byers S. Derrick-Roberts AL, et al. Among authors: pyragius ce. Hum Gene Ther. 2014 Sep;25(9):798-810. doi: 10.1089/hum.2013.141. Epub 2014 Aug 14. Hum Gene Ther. 2014. PMID: 25003807
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