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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Detalles Bibliográficos
Autores principales: Liu, Lin, Lee, Wang-Sik, Doray, Balraj, Kornfeld, Stuart
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2017
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5415318/
https://www.ncbi.nlm.nih.gov/pubmed/28480305
http://dx.doi.org/10.1016/j.omtm.2017.03.006

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5415318/
https://www.ncbi.nlm.nih.gov/pubmed/28480305
http://dx.doi.org/10.1016/j.omtm.2017.03.006

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