Hay Mele, B.a , Citro, V.b , Andreotti, G.c , Cubellis, M.V.b
Drug repositioning can accelerate discovery of pharmacological chaperones
(2015) Orphanet Journal of Rare Diseases, 10 (1), art. no. 10, .
https://www.scopus.com/inward/record.uri?eid=2-s2.0-84976402620&partnerID=40&md5=3516171793496d8f0d57eb789fc7165f
DOI: 10.1186/s13023-015-0273-2
AFFILIATIONS: aDepartment of Agricultural and Food Sciences, University Federico II, Naples, Italy;
bDepartment of Biology, University Federico II, Naples, Italy;
Istituto di Chimica Biomolecolare -CNR, Pozzuoli, Italy
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. © 2015 Hay Mele et al.; licensee BioMed Central.
AUTHOR KEYWORDS: Drug repositioning; Pharmacological chaperone
DOCUMENT TYPE: Article
