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...

Descripción completa

Detalles Bibliográficos
Autores principales: Hordeaux, Juliette, Hinderer, Christian, Goode, Tamara, 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/PMC6070702/
https://www.ncbi.nlm.nih.gov/pubmed/30073178
http://dx.doi.org/10.1016/j.omtm.2018.06.004

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6070702/
https://www.ncbi.nlm.nih.gov/pubmed/30073178
http://dx.doi.org/10.1016/j.omtm.2018.06.004

Ejemplares similares