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Drug repositioning can accelerate discovery of pharmacological chaperones

A promising strategy for the treatment of genetic diseases, pharmacological chaperone therapy, has been proposed recently. It exploits small molecules which can be administered orally, reach difficult tissues such as the brain and have low cost. This strategy has a vast field of application. In orde...

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Detalhes bibliográficos
Publicado no:Orphanet J Rare Dis
Main Authors: Hay Mele, Bruno, Citro, Valentina, Andreotti, Giuseppina, Cubellis, Maria Vittoria
Formato: Artigo
Idioma:Inglês
Publicado em: BioMed Central 2015
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4429356/
https://ncbi.nlm.nih.gov/pubmed/25947946
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s13023-015-0273-2
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