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RTB lectin-mediated delivery of lysosomal α-L-iduronidase mitigates disease manifestations systemically including the central nervous system
Mucopolysaccharidosis type I (MPS I) is a lysosomal disease resulting from deficiency in the α-L-iduronidase (IDUA) hydrolase and subsequent accumulation of glycosaminoglycan (GAG). Clinically, enzyme replacement therapy (ERT) with IDUA achieves negligible neurological benefits presumably due to blo...
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| Опубликовано в: : | Mol Genet Metab |
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| Главные авторы: | , , , |
| Формат: | Artigo |
| Язык: | Inglês |
| Опубликовано: |
2017
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| Предметы: | |
| Online-ссылка: | https://ncbi.nlm.nih.gov/pmc/articles/PMC5808854/ https://ncbi.nlm.nih.gov/pubmed/29198892 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymgme.2017.11.013 |
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