AAVrh10 Vector Corrects Disease Pathology in MPS IIIA Mice and Achieves Widespread Distribution of SGSH in Large Animal Brains

Patients with mucopolysaccharidosis type IIIA (MPS IIIA) lack the lysosomal enzyme sulfamidase (SGSH), which is responsible for the degradation of heparan sulfate (HS). Build-up of undegraded HS results in severe progressive neurodegeneration for which there is currently no treatment. The ability of...

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Publicado en:Mol Ther Methods Clin Dev
Main Authors: Hocquemiller, Michaël, Hemsley, Kim M., Douglass, Meghan L., Tamang, Sarah J., Neumann, Daniel, King, Barbara M., Beard, Helen, Trim, Paul J., Winner, Leanne K., Lau, Adeline A., Snel, Marten F., Gomila, Cathy, Ausseil, Jérôme, Mei, Xin, Giersch, Laura, Plavsic, Mark, Laufer, Ralph
Formato: Artigo
Idioma:Inglês
Publicado: American Society of Gene & Cell Therapy 2019
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Article
Acceso en liña:https://ncbi.nlm.nih.gov/pmc/articles/PMC6940615/
https://ncbi.nlm.nih.gov/pubmed/31909089
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2019.12.001
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