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Novel Patient Cell-Based HTS Assay for Identification of Small Molecules for a Lysosomal Storage Disease
Small molecules have been identified as potential therapeutic agents for lysosomal storage diseases (LSDs), inherited metabolic disorders caused by defects in proteins that result in lysosome dysfunctional. Some small molecules function assisting the folding of mutant misfolded lysosomal enzymes tha...
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| Hoofdauteurs: | , , , , , , , , |
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| Formaat: | Artigo |
| Taal: | Inglês |
| Gepubliceerd in: |
Public Library of Science
2011
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| Onderwerpen: | |
| Online toegang: | https://ncbi.nlm.nih.gov/pmc/articles/PMC3244463/ https://ncbi.nlm.nih.gov/pubmed/22216298 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1371/journal.pone.0029504 |
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