Lysosomal Storage Disease
New transgenic mice provide a way to investigate glucuronidase deficiency
Published in Gene Therapy Weekly, June 26th, 2003
"Mucopolysaccharidosis VII (MPS VII, Sly syndrome) is an autosomal recessive lysosomal storage disease caused by P-glucuronidase (GUS) deficiency. A naturally occurring mouse model of that disease has been very useful for studying experimental, approaches to therapy. However, immune responses can complicate evaluation of the long-term benefits of enzyme replacement or gene therapy delivered to adult MPS VII mice," scientists in the United States report.
"To make this model useful for studying the long-term effectiveness and side effects of experimental therapies delivered to...
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Source: Gene Therapy Weekly (2003-06-26)
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