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31. 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. Hum Gene Ther; 2014 Sep; 25(9):798-810. PubMed ID: 25003807 [Abstract] [Full Text] [Related]
32. Brain transplantation of genetically modified bone marrow stromal cells corrects CNS pathology and cognitive function in MPS VII mice. Sakurai K, Iizuka S, Shen JS, Meng XL, Mori T, Umezawa A, Ohashi T, Eto Y. Gene Ther; 2004 Oct; 11(19):1475-81. PubMed ID: 15295619 [Abstract] [Full Text] [Related]
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