Article
Adenovirus-mediated gene therapy for mucopolysaccharidosis VII: involvement of cross-correction in wide-spread distribution of the gene products and long-term effects of CTLA-4Ig coexpression.
Molecular therapy : the journal of the American Society of Gene Therapy - 1 May 2000
Kosuga M, Takahashi S, Sasaki K, Li X K, Fujino M, Hamada H, Suzuki S, Yamada M, Matsuo N, Okuyama T
Abstract excerpt
Recombinant adenoviruses expressing human beta-glucuronidase (AxCAhGUS) and CTLA-4Ig (AxCACTLA-4Ig) were generated and therapeutic efficacy was investigated using a murine model of mucopolysaccharidosis type VII (MPSVII). Seven days after the intravenous administration of AxCAhGUS, high levels of beta-glucuronidase (GUSB) activity were observed in the liver, spleen, heart, lung, kidney, and serum, while viral DNA...
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