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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Autori principali: Conn, P. Michael, Smithson, David C., Hodder, Peter S., Stewart, M. David, Behringer, Richard R., Smith, Emery, Ulloa-Aguirre, Alfredo, Janovick, Jo Ann
Natura: Artigo
Lingua:Inglês
Pubblicazione: 2013
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Article
Accesso online: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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