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Intravenous delivery for treatment of mucopolysaccharidosis type I: A comparison of AAV serotypes 9 and rh10

Mucopolysaccharidosis type I (MPS I) is an inherited metabolic disorder caused by deficiency of alpha-L-iduronidase (IDUA), resulting in accumulation of heparan and dermatan sulfate glycosaminoglycans (GAGs). Individuals with the most severe form of the disease (Hurler syndrome) suffer from neurodeg...

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Detalles Bibliográficos
Autores principales:	Belur, Lalitha R., Podetz-Pedersen, Kelly M., Tran, Thuy An, Mesick, Joshua A., Singh, Nathaniel M., Riedl, Maureen, Vulchanova, Lucy, Kozarsky, Karen F., McIvor, R. Scott
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Elsevier 2020
Materias:	Research Paper
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7242863/ https://www.ncbi.nlm.nih.gov/pubmed/32461912 http://dx.doi.org/10.1016/j.ymgmr.2020.100604

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