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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Detalhes bibliográficos
Publicado no:Mol Genet Metab Rep
Main Authors: 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: Artigo
Idioma:Inglês
Publicado em: Elsevier 2020
Assuntos:
Research Paper
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC7242863/
https://ncbi.nlm.nih.gov/pubmed/32461912
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymgmr.2020.100604
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