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Transitioning Pharmacoperones to Therapeutic Use: In Vivo Proof-of-Principle and Design of High Throughput Screens
A pharmacoperone (from “pharmacological chaperone”) is a small molecule that enters cells and serves as molecular scaffolding in order to cause otherwise-misfolded mutant proteins to fold and route correctly within the cell. Pharmacoperones have broad therapeutic applicability since a large number o...
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| Auteurs principaux: | , , , , , , , |
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| Format: | Artigo |
| Langue: | Inglês |
| Publié: |
2013
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| Sujets: | |
| Accès en ligne: | https://ncbi.nlm.nih.gov/pmc/articles/PMC4047138/ https://ncbi.nlm.nih.gov/pubmed/24373832 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.phrs.2013.12.004 |
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