Correction of Hunter syndrome in the MPSII mouse model by AAV2/8-mediated gene delivery
Mucopolysaccharidosis type II (MPSII; Hunter syndrome) is a lysosomal storage disorder caused by a deficiency in the enzyme iduronate 2-sulfatase (IDS). At present, the therapeutic approaches for MPSII are enzyme replacement therapy and bone marrow transplantation, although these therapies have some...
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| Autors principals: | , , , , , , , |
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| Format: | Artigo |
| Idioma: | anglès |
| Publicat: |
2006
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| Accés en línia: | https://doi.org/10.1093/hmg/ddl038 https://academic.oup.com/hmg/article-pdf/15/7/1225/17243398/ddl038.pdf |
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