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Brain‐targeted stem cell gene therapy corrects mucopolysaccharidosis type II via multiple mechanisms
The pediatric lysosomal storage disorder mucopolysaccharidosis type II is caused by mutations in IDS, resulting in accumulation of heparan and dermatan sulfate, causing severe neurodegeneration, skeletal disease, and cardiorespiratory disease. Most patients manifest with cognitive symptoms, which ca...
Αποθηκεύτηκε σε:
| Τόπος έκδοσης: | EMBO Mol Med |
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| Κύριοι συγγραφείς: | , , , , , , , , |
| Μορφή: | Artigo |
| Γλώσσα: | Inglês |
| Έκδοση: |
John Wiley and Sons Inc.
2018
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| Θέματα: | |
| Διαθέσιμο Online: | https://ncbi.nlm.nih.gov/pmc/articles/PMC6034129/ https://ncbi.nlm.nih.gov/pubmed/29884617 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.15252/emmm.201708730 |
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