An immune deficient mouse model for mucopolysaccharidosis IIIA (Sanfilippo syndrome)
An immune deficient mouse model for mucopolysaccharidosis IIIA (Sanfilippo syndrome)
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London: Nature Publishing Group UK
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English
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Publisher
London: Nature Publishing Group UK
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Mucopolysaccharidosis III (MPSIII, Sanfilippo syndrome) is a devastating lysosomal storage disease that primarily affects the central nervous system. MPSIIIA is caused by loss-of-function mutations in the gene coding for sulfamidase (N-sulfoglucosamine sulfohydrolase/SGSH) resulting in SGSH enzyme deficiency, a buildup of heparin sulfate and subseq...
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Full title
An immune deficient mouse model for mucopolysaccharidosis IIIA (Sanfilippo syndrome)
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TN_cdi_doaj_primary_oai_doaj_org_article_71342b495ee445a7a75611d2c25d3aa4
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https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_doaj_primary_oai_doaj_org_article_71342b495ee445a7a75611d2c25d3aa4
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ISSN
2045-2322
E-ISSN
2045-2322
DOI
10.1038/s41598-023-45178-0
