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Therapeutic Rescue of Misfolded Mutants: Validation of Primary High Throughput Screens for Identification of Pharmacoperone Drugs

BACKGROUND: Functional rescue of misfolded mutant receptors by small non-peptide molecules has been demonstrated. These small, target-specific molecules (pharmacological chaperones or “pharmacoperones”) serve as molecular templates, promote correct folding and allow otherwise misfolded mutants to pa...

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Autors principals: Janovick, Jo Ann, Park, Byung S., Conn, P. Michael
Format: Artigo
Idioma:Inglês
Publicat: Public Library of Science 2011
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Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC3144936/
https://ncbi.nlm.nih.gov/pubmed/21818389
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1371/journal.pone.0022784
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