title: Drug repositioning can accelerate discovery of pharmacological chaperones.
abstract: 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 order to make drug development as fast as possible, it is important to exploit drug repositioning. We evaluated the impact and limitations of this approach for rare diseases and we provide a shortcut in finding drugs for off-target usage.
authors.affiliation: Department of Agricultural and Food Sciences, University Federico II, Naples, Italy. bruno.haymele@unina.it.
authors.forename: Bruno
authors.initials: B
authors.lastname: Hay Mele
authors.affiliation: Department of Biology, University Federico II, Naples, Italy. vale.ctr@gmail.com.
authors.forename: Valentina
authors.initials: V
authors.lastname: Citro
authors.affiliation: Istituto di Chimica Biomolecolare -CNR, Pozzuoli, Italy. giuseppina.andreotti@icb.cnr.it.
authors.forename: Giuseppina
authors.initials: G
authors.lastname: Andreotti
authors.affiliation: Department of Biology, University Federico II, Naples, Italy. cubellis@unina.it.
authors.forename: Maria Vittoria
authors.initials: MV
authors.lastname: Cubellis
mesh_terms: D017277:Drug Approval; D058492:Drug Repositioning; D030342:Genetic Diseases, Inborn / Q000188:drug therapy*; D006801:Humans