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Clearance of heparan sulfate in the brain prevents neurodegeneration and neurocognitive impairment in MPS II mice
Mucopolysaccharidosis II (MPS II), a lysosomal storage disease caused by mutations in iduronate-2-sulfatase (IDS), is characterized by a wide variety of somatic and neurologic symptoms. The currently approved intravenous enzyme replacement therapy with recombinant IDS (idursulfase) is ineffective fo...
Αποθηκεύτηκε σε:
| Τόπος έκδοσης: | Mol Ther |
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| Κύριοι συγγραφείς: | , , , , , , , , , , , |
| Μορφή: | Artigo |
| Γλώσσα: | Inglês |
| Έκδοση: |
American Society of Gene & Cell Therapy
2021
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| Θέματα: | |
| Διαθέσιμο Online: | https://ncbi.nlm.nih.gov/pmc/articles/PMC8116601/ https://ncbi.nlm.nih.gov/pubmed/33508431 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2021.01.027 |
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