Engineering of GlcNAc-1-Phosphotransferase for Production of Highly Phosphorylated Lysosomal Enzymes for Enzyme Replacement Therapy

Several lysosomal enzymes currently used for enzyme replacement therapy in patients with lysosomal storage diseases contain very low levels of mannose 6-phosphate, limiting their uptake via mannose 6-phosphate receptors on the surface of the deficient cells. These enzymes are produced at high levels...

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Publicado en:Mol Ther Methods Clin Dev
Autores principales: Liu, Lin, Lee, Wang-Sik, Doray, Balraj, Kornfeld, Stuart
Formato: Artigo
Lenguaje:Inglês
Publicado: American Society of Gene & Cell Therapy 2017
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Original Article
Acceso en línea:https://ncbi.nlm.nih.gov/pmc/articles/PMC5415318/
https://ncbi.nlm.nih.gov/pubmed/28480305
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2017.03.006
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