Intranasal Adeno-Associated Virus Mediated Gene Delivery and Expression of Human Iduronidase in the Central Nervous System: A Noninvasive and Effective Approach for Prevention of Neurologic Disease in Mucopolysaccharidosis Type I

Mucopolysaccharidosis type I (MPS I) is a progressive, multi-systemic, inherited metabolic disease caused by deficiency of α-L-iduronidase (IDUA). Current treatments for this disease are ineffective in treating central nervous system (CNS) disease due to the inability of lysosomal enzymes to travers...

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Veröffentlicht in:Hum Gene Ther
Hauptverfasser: Belur, Lalitha R., Temme, Alexa, Podetz-Pedersen, Kelly M., Riedl, Maureen, Vulchanova, Lucy, Robinson, Nicholas, Hanson, Leah R., Kozarsky, Karen F., Orchard, Paul J., Frey, William H., Low, Walter C., McIvor, R. Scott
Format: Artigo
Sprache:Inglês
Veröffentlicht: Mary Ann Liebert, Inc. 2017
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Research Articles
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC5549804/
https://ncbi.nlm.nih.gov/pubmed/28462595
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2017.187
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