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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Detalles Bibliográficos
Autores principales: Hordeaux, Juliette, Hinderer, Christian, Goode, Tamara, Katz, Nathan, Buza, Elizabeth L., Bell, Peter, Calcedo, Roberto, Richman, Laura K., Wilson, James M.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2018
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6070681/
https://www.ncbi.nlm.nih.gov/pubmed/30073179
http://dx.doi.org/10.1016/j.omtm.2018.06.003

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