Targeting Root Cause by Systemic scAAV9-hIDS Gene Delivery: Functional Correction and Reversal of Severe MPS II in Mice

No treatment is available to address the neurological need and reversibility of MPS II. We developed a scAAV9-hIDS vector to deliver the human iduronate-2-sulfatase gene and test it in mouse model. We treated MPS II mice at different disease stages with an intravenous injection of scAAV9-mCMV-hIDS a...

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Detalles Bibliográficos
Autores principales: Fu, Haiyan, Zaraspe, Kim, Murakami, Naoko, Meadows, Aaron S., Pineda, Ricardo J., McCarty, Douglas M., Muenzer, Joseph
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/PMC6125796/
https://www.ncbi.nlm.nih.gov/pubmed/30191159
http://dx.doi.org/10.1016/j.omtm.2018.07.005

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6125796/
https://www.ncbi.nlm.nih.gov/pubmed/30191159
http://dx.doi.org/10.1016/j.omtm.2018.07.005

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