Gene therapy for cross-correction of somatic organs and the CNS in mucopolysaccharidosis II in rodents and non-human primates

Mucopolysaccharidosis II (MPS II) is a rare lysosomal storage disease characterized by deficient activity of iduronate-2-sulfatase (I2S), leading to pathological accumulation of glycosaminoglycans (GAGs) in tissues. We used iduronate-2-sulfatase knockout (Ids KO) mice to investigate if liver-directe...

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Detalles Bibliográficos
Autores principales: Chen, Nancy, Ehmann, David E., Crooker, Robert, Derakhchan, Katayoun, Fang, Xiaodong, Felice, Brian, Galbreath, Elizabeth J., Glaus, Charles, Gu, Hongbo, Huang, Yan, Li, Christine, Li, Xing, Liu, Nan, Palmieri, Kathleen, Simic, Damir, Sypek, Joseph, Thompson, Susan, Winkelmann, Christopher T., Choi, Vivian W.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2023
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10285185/
https://www.ncbi.nlm.nih.gov/pubmed/37359415
http://dx.doi.org/10.1016/j.omtm.2023.03.014

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10285185/
https://www.ncbi.nlm.nih.gov/pubmed/37359415
http://dx.doi.org/10.1016/j.omtm.2023.03.014

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