Adenovirus-mediated gene transfer and expression of human β-glucuronidase gene in the liver, spleen, and central nervous system in mucopolysaccharidosis type VII mice

Mucopolysaccharidosis type VII (Sly syndrome) is a lysosomal storage disease caused by inherited deficiency of the lysosomal enzyme β-glucuronidase. A murine model of this disorder has been well characterized and used to study a number of forms of experimental therapies, including gene therapy. We p...

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Hlavní autoři: Ohashi, Toya, Watabe, Kazuhiko, Uehara, Keiko, Sly, William S., Vogler, Carole, Eto, Yoshikatsu
Médium: Artigo
Jazyk:Inglês
Vydáno: The National Academy of Sciences of the USA 1997
Témata:
Biological Sciences
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC19783/
https://ncbi.nlm.nih.gov/pubmed/9037045
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