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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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書目詳細資料
發表在:Sci Adv
Main Authors: 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.
格式: Artigo
語言:Inglês
出版: American Association for the Advancement of Science 2020
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在線閱讀:https://ncbi.nlm.nih.gov/pmc/articles/PMC7732195/
https://ncbi.nlm.nih.gov/pubmed/33310843
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1126/sciadv.abb0385
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