CNS-directed gene therapy for the treatment of neurologic and somatic mucopolysaccharidosis type II (Hunter syndrome)

Mucopolysaccharidosis type II (MPSII) is an X-linked lysosomal storage disease characterized by severe neurologic and somatic disease caused by deficiency of iduronate-2-sulfatase (IDS), an enzyme that catabolizes the glycosaminoglycans heparan and dermatan sulphate. Intravenous enzyme replacement t...

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Enregistré dans:
Détails bibliographiques
Publié dans:JCI Insight
Auteurs principaux: Motas, Sandra, Haurigot, Virginia, Garcia, Miguel, Marcó, Sara, Ribera, Albert, Roca, Carles, Sánchez, Xavier, Sánchez, Víctor, Molas, Maria, Bertolin, Joan, Maggioni, Luca, León, Xavier, Ruberte, Jesús, Bosch, Fatima
Format: Artigo
Langue:Inglês
Publié: American Society for Clinical Investigation 2016
Sujets:
Research Article
Accès en ligne:https://ncbi.nlm.nih.gov/pmc/articles/PMC5033872/
https://ncbi.nlm.nih.gov/pubmed/27699273
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1172/jci.insight.86696
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