Exploiting the diphtheria toxin internalization receptor enhances delivery of proteins to lysosomes for enzyme replacement therapy

Enzyme replacement therapy, in which a functional copy of an enzyme is injected either systemically or directly into the brain of affected individuals, has proven to be an effective strategy for treating certain lysosomal storage diseases. The inefficient uptake of recombinant enzymes via the mannos...

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Detalles Bibliográficos
Autores principales: Sugiman-Marangos, S. N., Beilhartz, G. L., Zhao, X., Zhou, D., Hua, R., Kim, P. K., Rini, J. M., Minassian, B. A., Melnyk, R. A.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Association for the Advancement of Science 2020
Materias:
Research Articles
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7732195/
https://www.ncbi.nlm.nih.gov/pubmed/33310843
http://dx.doi.org/10.1126/sciadv.abb0385

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7732195/
https://www.ncbi.nlm.nih.gov/pubmed/33310843
http://dx.doi.org/10.1126/sciadv.abb0385

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