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November 1, 1999Experimental NeurologyOpen Access

Sustained Production of β-Glucuronidase from Localized Sites after AAV Vector Gene Transfer Results in Widespread Distribution of Enzyme and Reversal of Lysosomal Storage Lesions in a Large Volume of Brain in Mucopolysaccharidosis VII Mice

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Authors

ASAnnette F. SkorupaThe Wistar InstituteKFKrisna J. FisherHuman Gene Therapy Research InstituteJames M. WilsonJames M. WilsonThe Wistar Institute

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Skorupa et al. (1999) studied this question.

synapsesocial.com/papers/6a6f26786c240de38cdb66c8https://doi.org/10.1006/exnr.1999.7176
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Also Consider

Synapse has enriched 5 closely related papers on similar clinical questions. Consider them for comparative context:

  1. 1Dopaminergic Neurons Protected from Degeneration by GDNF Gene Therapy1997 · 631 citations
  2. 2Murine mucopolysaccharidosis type VII. Characterization of a mouse with beta-glucuronidase deficiency.1989 · 291 citations
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  4. 4Increased life span and correction of metabolic defects in murine mucopolysaccharidosis type VII after syngeneic bone marrow transplantation1991 · 184 citations
  5. 5Selective and Rapid Uptake of Adeno-Associated Virus Type 2 in Brain1998 · 302 citations