Toxicology Study of Intra-Cisterna Magna Adeno-Associated Virus 9 Expressing Human Alpha-L-Iduronidase in Rhesus Macaques

Mucopolysaccharidosis type I is a recessive genetic disease caused by deficiency of the lysosomal enzyme α-L-iduronidase, which leads to a neurodegenerative and systemic disease called Hurler syndrome in its most severe form. Several clinical trials are evaluating adeno-associated virus serotype 9 (...

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Hlavní autoři:	Juliette Hordeaux, Christian Hinderer, Tamara Goode, Nathan Katz, Elizabeth L. Buza, Peter Bell, Roberto Calcedo, Laura K. Richman, James M. Wilson
Médium:	Artigo
Jazyk:	Inglês
Vydáno:	Elsevier 2018-09-01
Edice:	Molecular Therapy: Methods & Clinical Development
On-line přístup:	http://www.sciencedirect.com/science/article/pii/S2329050118300573
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