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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...
Autores principales: | , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Public Library of Science
2011
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3144936/ https://www.ncbi.nlm.nih.gov/pubmed/21818389 http://dx.doi.org/10.1371/journal.pone.0022784 |