854. Feasibility of AAV-Mediated Gene Therapy Examined Using a New Murine Model (D409V/null) of Gauc...
854. Feasibility of AAV-Mediated Gene Therapy Examined Using a New Murine Model (D409V/null) of Gaucher Disease
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Milwaukee: Elsevier Inc
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Language
English
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Milwaukee: Elsevier Inc
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Contents
Gaucher disease is the most common of the more than 40 currently described lysosomal storage diseases. It is caused by mutations in the gene encoding the lysosomal hydrolase, glucocerebrosidase (GC). The loss or deficiency of this enzyme results in the accumulation of the substrate, glucosylceramide (GL-1), in tissue macrophages primarily of the li...
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854. Feasibility of AAV-Mediated Gene Therapy Examined Using a New Murine Model (D409V/null) of Gaucher Disease
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TN_cdi_proquest_journals_1793391490
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https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_proquest_journals_1793391490
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ISSN
1525-0016
E-ISSN
1525-0024
DOI
10.1016/j.ymthe.2004.06.762
