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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Autores principales: Hay Mele, Bruno, Citro, Valentina, Andreotti, Giuseppina, Cubellis, Maria Vittoria
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2015
Materias:
Letter to the Editor
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4429356/
https://www.ncbi.nlm.nih.gov/pubmed/25947946
http://dx.doi.org/10.1186/s13023-015-0273-2
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author Hay Mele, Bruno
Citro, Valentina
Andreotti, Giuseppina
Cubellis, Maria Vittoria
author_facet Hay Mele, Bruno
Citro, Valentina
Andreotti, Giuseppina
Cubellis, Maria Vittoria
author_sort Hay Mele, Bruno
collection PubMed
description 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. ELECTRONIC SUPPLEMENTARY MATERIAL: The online version of this article (doi:10.1186/s13023-015-0273-2) contains supplementary material, which is available to authorized users.
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spelling pubmed-44293562015-05-14 Drug repositioning can accelerate discovery of pharmacological chaperones Hay Mele, Bruno Citro, Valentina Andreotti, Giuseppina Cubellis, Maria Vittoria Orphanet J Rare Dis Letter to the Editor 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. ELECTRONIC SUPPLEMENTARY MATERIAL: The online version of this article (doi:10.1186/s13023-015-0273-2) contains supplementary material, which is available to authorized users. BioMed Central 2015-05-07 /pmc/articles/PMC4429356/ /pubmed/25947946 http://dx.doi.org/10.1186/s13023-015-0273-2 Text en © Hay Mele et al.; licensee BioMed Central. 2015 This is an Open Access article distributed under the terms of the Creative Commons Attribution License (http://creativecommons.org/licenses/by/4.0), which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly credited. The Creative Commons Public Domain Dedication waiver (http://creativecommons.org/publicdomain/zero/1.0/) applies to the data made available in this article, unless otherwise stated.
spellingShingle Letter to the Editor
Hay Mele, Bruno
Citro, Valentina
Andreotti, Giuseppina
Cubellis, Maria Vittoria
Drug repositioning can accelerate discovery of pharmacological chaperones
title Drug repositioning can accelerate discovery of pharmacological chaperones
title_full Drug repositioning can accelerate discovery of pharmacological chaperones
title_fullStr Drug repositioning can accelerate discovery of pharmacological chaperones
title_full_unstemmed Drug repositioning can accelerate discovery of pharmacological chaperones
title_short Drug repositioning can accelerate discovery of pharmacological chaperones
title_sort drug repositioning can accelerate discovery of pharmacological chaperones
topic Letter to the Editor
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4429356/
https://www.ncbi.nlm.nih.gov/pubmed/25947946
http://dx.doi.org/10.1186/s13023-015-0273-2
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