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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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Detalhes bibliográficos
Publicado no:Mol Genet Metab
Principais autores: Ou, Li, Przybilla, Michael J, Koniar, Brenda, Whitley, Chester B
Formato: Artigo
Idioma:Inglês
Publicado em: 2017
Assuntos:
Acesso em linha: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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