Guanidinylated Neomycin Conjugation Enhances Intranasal Enzyme Replacement in the Brain

Iduronidase (IDUA)-deficient mice accumulate glycosaminoglycans in cells and tissues and exhibit many of the same neuropathological symptoms of patients suffering from Mucopolysaccharidosis I. Intravenous enzyme-replacement therapy for Mucopolysaccharidosis I ameliorates glycosaminoglycan storage an...

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Pubblicato in:Mol Ther
Autori principali: Tong, Wenyong, Dwyer, Chrissa A., Thacker, Bryan E., Glass, Charles A., Brown, Jillian R., Hamill, Kristina, Moremen, Kelley W., Sarrazin, Stéphane, Gordts, Philip L.S.M., Dozier, Lara E., Patrick, Gentry N., Tor, Yitzhak, Esko, Jeffrey D.
Natura: Artigo
Lingua:Inglês
Pubblicazione: American Society of Gene & Cell Therapy 2017
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Original Article
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC5768556/
https://ncbi.nlm.nih.gov/pubmed/28958576
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2017.08.007
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