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.

Drug repositioning can accelerate discovery of pharmacological chaperones / B. Hay Mele, V. Citro, G. Andreotti, M.V. Cubellis. - In: ORPHANET JOURNAL OF RARE DISEASES. - ISSN 1750-1172. - 10(2015), pp. 55.1-55.3. [10.1186/s13023-015-0273-2]

Drug repositioning can accelerate discovery of pharmacological chaperones

V. Citro;
2015

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.
Pharmacological chaperone; Drug repositioning
Settore BIO/10 - Biochimica
Settore MED/04 - Patologia Generale
Settore BIO/11 - Biologia Molecolare
Settore BIO/13 - Biologia Applicata
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Utilizza questo identificativo per citare o creare un link a questo documento: http://hdl.handle.net/2434/724702
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