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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Bibliographic Details
Main Authors: Hay Mele, Bruno, Citro, Valentina, Andreotti, Giuseppina, Cubellis, Maria Vittoria
Format: Online
Language:English
Published: BioMed Central 2015
Online Access:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4429356/