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Toxicology Study of Intra-Cisterna Magna Adeno-Associated Virus 9 Expressing Iduronate-2-Sulfatase in Rhesus Macaques

Hunter syndrome is an X-linked recessive disease caused by deficiency of the lysosomal enzyme iduronate-2-sulfatase. The severe form of this progressive, systemic, and neurodegenerative disease results in loss of cognitive skills and early death. Several clinical trials are evaluating adeno-associat...

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Detalhes bibliográficos
Publicado no:Mol Ther Methods Clin Dev
Main Authors: Hordeaux, Juliette, Hinderer, Christian, Goode, Tamara, Buza, Elizabeth L., Bell, Peter, Calcedo, Roberto, Richman, Laura K., Wilson, James M.
Formato: Artigo
Idioma:Inglês
Publicado em: American Society of Gene & Cell Therapy 2018
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC6070702/
https://ncbi.nlm.nih.gov/pubmed/30073178
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2018.06.004
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